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Monthly News Roundup - December 2024

Medically reviewed by Leigh Ann Anderson, PharmD. Last updated on Dec 31, 2024.

FDA Approves Zepbound as the First Prescription Drug for Obstructive Sleep Apnea in Adults with Obesity

On December 20, 2024, the U.S. Food and Drug Administration (FDA) approved Eli Lilly’s Zepbound (tirzepatide) for treatment of moderate-to-severe obstructive sleep apnea (OSA) in adults with obesity. It is the first and only prescription medicine for this indication. In addition to improved OSA, on average, patients also lost 45 to 50 lbs during treatment.

• Obstructive sleep apnea (OSA) is a serious breathing disorder characterized by complete or partial collapses of the upper airway during sleep. This can lead to pauses in breathing and shallow breathing, waking from sleep and a potential decrease in blood oxygen saturation. Snoring, fatigue and excessive daytime sleepiness can be key symptoms and may occur in patients with obesity.
• Zepbound is a glucose-dependent insulinotropic polypeptide (GIP) receptor and glucagon-like peptide-1 (GLP-1) receptor agonist also approved to reduce excess body weight and maintain long-term weight reduction in adults with obesity or with overweight and at least one weight-related medical condition like high blood pressure, high cholesterol or heart disease.
• The recommended maintenance dose for OSA is 10 mg or 15 mg injected subcutaneously once weekly, after initial dose escalation to moderate stomach side effects.
• Approval was based on the SURMOUNT-OSA Phase 3 studies with 469 participants. Zepbound was evaluated for the treatment of OSA in adults with obesity, with and without positive airway pressure (PAP) therapy over one year.
• The primary objective was to demonstrate that Zepbound was superior in change in apnea-hypopnea index (AHI) from baseline at 52 weeks as compared to placebo (an injection with no medicine). The apnea-hypopnea index (AHI) measures the severity of OSA and looks at the number of times per hour during sleep that breathing stops (apnea) or becomes significantly reduced (hypopnea).
• Results in patients not using PAP showed that Zepbound led to 25 fewer breathing disruptions per hour compared to 5 disruptions with placebo. With PAP, Zepbound led to 29 fewer breathing disruptions per hour compared to 6 disruptions with placebo. After one year, 42% or 50% of adults on Zepbound experienced remission or mild, non-symptomatic OSA, compared to 16% or 14% on placebo.
• In addition to improved OSA symptoms, adults on Zepbound lost an average of 45 lbs (18%) of their body weight, while adults on Zepbound and PAP therapy lost an average of 50 lbs (20%) of their body weight, compared to 4 lbs (2%) and 6 lbs (2%) on placebo, respectively.
• Common adverse reactions include nausea, diarrhea, vomiting, constipation, abdominal pain, indigestion, injection site reactions, fatigue, hypersensitivity reactions, belching, hair loss, and heartburn. These are not all the possible side effects of Zepbound.
• Tirzepatide was first approved under the brand name Mounjaro (also from Lilly) in May 2022 to improve glycemic (blood sugar) control in adults with type 2 diabetes.

Gemtesa Cleared for Adult Males with Overactive Bladder Symptoms Also Treated for BPH

Gemtesa (vibegron) has been approved by the FDA to treat overactive bladder (OAB) with symptoms of urge urinary incontinence, urgency, and urinary frequency in adult males on pharmacological (drug) therapy for benign prostatic hyperplasia (BPH). Gemtesa is also approved to treat adults with overactive bladder symptoms of urge urinary incontinence, urgency, and urinary frequency.

• Gemtesa is classified as a beta-3 agonist and works by binding to this receptor to relax the bladder wall muscles (detrusor muscle) to increase how much the bladder can hold.
• It is taken as a once-daily tablet by mouth and can be taken with or without food.
• Approval was based on the results from the 12-week URO-901-3005 study, a Phase 3 trial of Gemtesa versus placebo over 24 weeks in approximately 1,100 men with overactive bladder (OAB) symptoms receiving pharmacological therapy for BPH. Results showed statistically significant reductions in the average number of micturition (urination) episodes per day and daily urgency episodes (sudden urge to urinate that is difficult to control) compared to placebo (pill with no medicine). In studies of OAB in adult males with BPH, hypertension (high blood pressure) and urinary tract infection were reported as side effects (≥2%) of patients.
• Warnings and precautions include urinary retention and angioedema of the face and/or larynx has been reported. Other common adverse reactions (≥2%) include headache, urinary tract infection, nasopharyngitis (common cold symptoms), diarrhea, nausea, and upper respiratory tract infection.
• Gemtesa, from Sumitomo Pharma, first received FDA approval on December 23, 2020 to treat adults with overactive bladder symptoms of urge urinary incontinence, urgency, and urinary frequency.

FDA Approves Opdivo Qvantig, the First Subcutaneously Injected PD-1 Inhibitor Cancer Immunotherapy

On December 27, 2024 the FDA granted approval for Opdivo Qvantig, a programmed death receptor-1 (PD-1)-blocking antibody combination of nivolumab and recombinant human hyaluronidase (rHuPH20). It is given to the patient over 3 to 5 minutes, on average, compared to an Opdivo 30-minute intravenous (IV) infusion.

• Subcutaneous administration may offer advantages over IV administration, including flexibility on treatment sites, fewer steps for preparation, and reduced administration time. Recombinant human hyaluronidase PH20 (rHuPH20), an enzyme, degrades hyaluronan (a sugar) in the area under the skin. This allows the medication to be rapidly dispersed and absorbed into the bloodstream when injected subcutaneously.
• Opdivo Qvantig immunotherapy can be used for most previously approved adult, solid tumor Opdivo indications as monotherapy (single) treatment, monotherapy maintenance following completion of Opdivo plus Yervoy (ipilimumab) combination therapy, or in combination with chemotherapy or cabozantinib. Indications include certain patients with renal cell (kidney) cancer, melanoma, non-small cell lung cancer, squamous cell cancer of the head and neck, urothelial (bladder) carcinoma, colorectal cancer, hepatocellular (liver) cancer, esophageal cancer, and gastric (stomach) cancer.
• Approval of Opdivo Qvantig is based on the results from the Phase 3 randomized, open-label CheckMate-67T trial, which demonstrated non-inferior co-primary pharmacokinetic (PK) exposures vs. IV Opdivo, similar efficacy in overall response rate (ORR), and a comparable safety profile vs. IV Opdivo.
• Opdivo Qvantig is for subcutaneous use only in the abdomen (stomach area) or thigh and is given by a healthcare provider. It has different dosage and administration instructions than intravenous nivolumab products. It is available as a single dose vial with 600 mg nivolumab and 10,000 units hyaluronidase per 5 mL (120 mg / 2,000 units per mL).
• Warnings and precautions include severe and fatal immune-mediated adverse reactions including pneumonitis, colitis, hepatitis and hepatotoxicity, among others. Serious side effects include pleural effusion, pneumonitis, hyperglycemia, hyperkalemia, hemorrhage and diarrhea.
• The most common adverse reactions (≥10%) in patients treated with Opdivo Qvantig were musculoskeletal pain (31%), fatigue (20%), itching (16%), rash (15%), low thyroid levels (12%), diarrhea (11%), cough (11%), and abdominal (stomach area) pain (10%).
• Opdivo Qvantig is manufactured by Bristol-Myers Squibb.

FDA Approves Vtama Cream for Atopic Dermatitis in People 2 Years of Age and Older

The FDA has cleared the topical use of steroid-free Vtama (tapinarof) in adults and pediatric patients 2 years of age and older with atopic dermatitis (eczema). Vtama is also approved for the topical treatment of plaque psoriasis in adults.

• Atopic dermatitis is a common, chronic, inflammatory skin disease characterized by persistent itch and recurrent skin lesions.
• Vtama is an aryl hydrocarbon receptor (AhR) modulating agent and is thought to work by activating a protein called AhR and via downregulation of pro-inflammatory cytokines, including interleukin 17. This action may help to reduce inflammation, itch, normalize skin barrier proteins and clear the skin.
• Vtama cream is applied to the affected areas once a day as a thin layer. It can be applied to all body areas including sensitive skin areas like your face, neck, armpits, chest/breasts, and groin but should not be applied to your eyes, mouth, or vagina.
• FDA approval was supported by the 8 week long ADORING studies, which showed a significant difference with Vtama in the number of patients achieving a score of "clear" or "almost clear" skin (Validated Investigator Global Assessment for AD) and a minimum 2-grade improvement from the start of the study when compared to the vehicle (cream with no medicine).
• Common adverse reactions (incidence ≥ 1%) in atopic dermatitis patients include upper respiratory tract infection, folliculitis (inflamed or infected hair follicles), lower respiratory tract infection, headache, asthma, vomiting, ear infection, pain in extremity and abdominal (stomach area) pain.
• Vtama is manufactured by Organon Pharmaceuticals and was approved for this use on Dec. 12, 2024.

FDA Approves Steqeyma, the Seventh Biosimilar to Stelara

Celltrion has announced the approval of Steqeyma (ustekinumab-stba) injection, a biosimilar to Stelara (ustekinumab). Steqeyma is used to treat adults and children 6 years of age and older with active psoriatic arthritis and moderate-to-severe plaque psoriasis, and adults with moderate-to-severe Crohn’s disease and ulcerative colitis.

• Steqeyma (ustekinumab-stba) is a fully human monoclonal antibody that selectively inhibits both interleukin (IL)-12 and IL-23, two cytokines that play an important role in inflammatory and immune responses.
• Steqeyma is not interchangeable with Stelara, meaning it cannot yet be substituted for the reference product (in this case, Stelara) by a pharmacist, depending upon state laws.
• Results from a Phase III study in adults with moderate to severe plaque psoriasis demonstrated that Steqeyma and Stelara are highly similar, and have no clinically meaningful differences in terms of safety and efficacy.
• Steqeyma can be given by subcutaneous injection or intravenous infusion.
• Serious warnings include increased infection risk, cancer risk, and allergic reactions, among others. Common adverse events may include nasopharyngitis, headache, fatigue, sinusitis and nausea, among others.
• Steqeyma, approved on Dec. 17, 2024, is the seventh FDA-approved Stelara biosimilar, following Yesintek, Imuldosa, Otulfi, Pyzchiva and Selarsdi in 2024, and Wezlana in 2023.

FDA Approves Liraglutide, the First Generic Referencing GLP-1 Agonist Victoza

The FDA has approved the first liraglutide generic referencing Victoza, a glucagon-like peptide-1 (GLP-1) receptor agonist injection indicated to improve glycemic (blood sugar) control in people 10 years and older with type 2 diabetes. It is used in addition to diet and exercise.

• Type 2 diabetes is a common chronic condition that occurs when the body does not use insulin well and cannot keep blood sugar at normal levels. Although usually diagnosed in adults, it is now increasingly diagnosed in children, teens and young adults. While generic liraglutide is only approved to treat type 2 diabetes, it may also help you lose some weight.
• Liraglutide improves blood sugar levels by acting like natural GLP-1 found in the body, which may be present in low levels in people living with type 2 diabetes. GLP-1 helps control blood sugar in type 2 diabetes by increasing insulin when needed, reducing glucose production in the liver, slowing digestion, and decreasing appetite.
• Liraglutide is administered subcutaneously (injected under the skin) once-daily at any time of day in the stomach area, thigh or upper arm. Patients or caregivers may self-administer injections after training. It is available as an 18 mg / 3 mL (6 mg / mL) pre-filled, single-patient-use pen that delivers doses of 0.6 mg, 1.2 mg, or 1.8 mg.
• Like Victoza, liraglutide carries a Boxed Warning for a risk of thyroid c-cell tumors and should not be used in patients with a personal or family history of medullary thyroid carcinoma (MTC) or in patients with Multiple Endocrine Neoplasia syndrome type 2 (MEN 2). Warnings and precautions include pancreatitis, gallbladder disease, acute kidney injury and pulmonary (lung) aspiration while under anesthesia, among others.
• Common adverse reactions in at least 5% of patients in studies included nausea, diarrhea, vomiting, decreased appetite, dyspepsia (heartburn, indigestion), constipation,
• The FDA granted the approval of generic liraglutide injection to Hikma Pharmaceuticals USA Inc. on December 23, 2024. Unlike Victoza, the liraglutide generic does not carry the indication to reduce the risk of major adverse cardiovascular events in adults with type 2 diabetes mellitus and established cardiovascular (heart) disease.
• Saxenda is another brand of liraglutide specifically approved for weight loss, in addition to diet and exercise, in at-risk patients with certain health conditions. Do not use Saxenda, Victoza or liraglutide at the same time.
• Liraglutide belongs to the drug class called glucagon-like peptide-1 (GLP-1) receptor agonists, which includes other medications such as Ozempic, Wegovy, and Ryebelsus.
• The FDA approved the first generic GLP-1 receptor agonist in November 2024 with the approval of a generic referencing Byetta (exenatide).

Alhemo Once-Daily Subcutaneous Injection Granted Approval for People with Hemophilia A or B with Inhibitors

Novo Nordisk announced on Dec. 20, 2024 the FDA-approval of Alhemo (concizumab-mtci) subcutaneous injection as a once-daily prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with hemophilia A or B with inhibitors.

• In hemophilia A or B, a rare disease, inhibitors can develop in some patients and lower the effectiveness of therapies. Treatment for patients in these cases to prevent bleeding is challenging and they often have few options.
• Alhemo is a tissue factor pathway inhibitor (TFPI) antagonist dosed in a prefilled, premixed pen for subcutaneous injection. It is not given as an IV infusion.
• Alhemo is designed to block a protein called TFPI in the body that stops blood from clotting. By blocking TFPI, the drug improves the production of thrombin, a protein that helps to clot the blood and prevent bleeding, when the other clotting factors are missing or deficient in the presence of inhibitors. This approval marks the first subcutaneous injection treatment of its kind for use in this patient population.
• Approval was based on the Phase 3 explorer7 study that compared the number of treated spontaneous and traumatic bleeding episodes, as measured by annual bleeding rate (ABR). Results showed an 86% reduction of ABR in patients who received Alhemo prophylaxis compared to no prophylaxis. The estimated mean ABR was 1.7 for patients on Alhemo prophylaxis compared to 11.8 for patients with no prophylaxis. The overall median ABR was zero for treated spontaneous and traumatic bleeds compared with 9.8 ABR in patients with no prophylaxis.
• Alhemo comes in a prefilled, premixed pen for subcutaneous injection (60 mg/1.5 mL, 150 mg/1.5 mL, or 300 mg/3 mL) given via a thin 32 gauge, 4 mm needle, which is provided separately. It may be self-administered or given by a caregiver in the abdomen (stomach area) or thigh after appropriate training, and when determined acceptable by a healthcare provider.
• Serious side effects may include blood clots and allergic reactions. The most common adverse reactions (in at least 5% of people) include injection site reactions and urticaria (hives).

Imfinzi Cleared as First Immunotherapy for Adults with Aggressive, Limited-Stage Small Cell Lung Cancer

The FDA has granted a new indication for Imfinzi (durvalumab) injection for use as a single agent to treat adult patients with limited-stage small cell lung cancer (LS-SCLC) whose disease has not progressed following concurrent platinum-based chemotherapy and radiation therapy.

• Small cell lung cancer is a highly aggressive form of lung cancer. The limited stage form (LS-SCLC) progresses rapidly, despite an initial treatment response to chemotherapy and radiation treatment. Only 15% to 30% of patients will be alive 5 years after diagnosis of LS-SCLC.
• Imfinzi, a PD-L1 checkpoint inhibitor, works by blocking the PD-L1 protein on the outside of cancer cells. This allows the immune system to more easily find and attack the cancer cells to help slow cancer growth and prolong survival.
• It is given as an intravenous (IV) infusion into a vein every 4 weeks, until disease progression, unacceptable toxicity, or a maximum of 24 months.
• Approval was based on significant findings from the ADRIATIC Phase III, placebo-controlled trial. In this study, Imfinzi reduced the risk of death by 27% vs. placebo with an estimated median overall survival (OS) of 55.9 months for Imfinzi compared to 33.4 months for placebo. In addition, Imfinzi also reduced the risk of disease progression or death by 24%, with median progression-free survival (PFS) of 16.6 months for Imfinzi vs. 9.2 months for placebo.
• No new safety signals were observed in the ADRIATIC trial. Warnings and precautions associated with Imfinzi include immune-mediated adverse reactions, infusion-related reactions, complications of stem cell treatment, and harm to an unborn baby.
• The most common treatment adverse reactions occurring in at least 20% of patients with limited-stage SCLC are pneumonitis or radiation pneumonitis (lung inflammation that affects breathing) and fatigue (tiredness).
• Imfinzi is also approved in combination with etoposide and either carboplatin or cisplatin, as first-line treatment of adult patients with extensive-stage small cell lung cancer (ES-SCLC), as well as for non-small cell lung cancer, biliary tract (bile duct and gallbladder) cancer, hepatocellular (liver) cancer, and endometrial cancer. It is manufactured by AstraZeneca and was approved for this use on Dec. 5, 2024.

Unloxcyt from Checkpoint Therapeutics Approved for Advanced Cutaneous Squamous Cell Skin Cancer

The FDA has cleared Unloxcyt (cosibelimab-ipdl), a programmed death ligand-1 (PD-L1) blocking antibody for the treatment of adults with metastatic or locally advanced cutaneous squamous cell carcinoma who are not candidates for surgery or radiation to cure the cancer.

• Unloxcyt is the first PD-L1 immune checkpoint inhibitor to be FDA-approved for this use. PD-L1, a protein, may be found on certain normal cells and on some types of cancer cells.
• Cutaneous squamous cell carcinoma (cSCC) is the second most common type of skin cancer in the U.S. with 1.8 million cases occurring annually. Most cases are localized and respond to surgery but about 40,000 cases are advanced and need further treatment. Risk factors for cSCC include chronic ultraviolet (UV) exposure and immunosuppression.
• Unloxcyt is a human immunoglobulin G1 (IgG1) monoclonal antibody that binds PD-L1 and blocks the interaction between PD-L1 and its T cell receptor proteins, PD-1 and B7.1. This allows the immune system to more easily find and attack the cancer cells to help slow cancer growth and prolong survival. Unloxcyt has also been shown to also induce antibody-dependent cell-mediated cytotoxicity (ADCC).
• The recommended dosage of Unloxcyt is 1,200 mg as an intravenous (IV) infusion over 60 minutes every 3 weeks.
• Approval was based on CK-301-101, an open-label study of Unloxcyt in 109 adults with metastatic CSCC (mCSCC) or locally advanced CSCC (laCSCC) who were not candidates for curative surgery or curative radiation. The primary endpoint was objective response rate (ORR) and duration of response (DOR). ORR was 47% (95% CI: 36, 59) for patients with metastatic cSCC (n=78) and 48% (95% CI: 30, 67) for patients with locally advanced cSCC (n=31), with 8% and 10% achieving a complete response, respectively. Median DOR was not reached (range: 1.4+, 34.1+) in patients with metastatic cSCC and was 17.7 months (range: 3.7+, 17.7) in patients with locally advanced cSCC.
• Warnings and precautions include immune-mediated adverse reactions, infusion-related reactions, Complications of Allogeneic Hematopoietic Stem Cell Transplantation (HSCT), and the potential for fetal harm.
• The most common adverse reactions (≥10%) were fatigue, musculoskeletal pain, rash, diarrhea, hypothyroidism (low thyroid levels), constipation, nausea, headache, pruritus (itching), edema (fluid build up), localized infection, and urinary tract infection (UTI).
• Unloxcyt is manufactured by Checkpoint Therapeutics and was approved on Dec. 13, 2024.

FDA Grants Approval to Bizengri for NRG1+ Pancreatic Cancer and Non-Small Cell Lung Cancer

On December 4, 2024 the FDA granted accelerated approval to Bizengri (zenocutuzumab-zbco) for treatment of patients with advanced, unresectable, or metastatic non-small cell lung cancer (NSCLC) or pancreatic cancer harboring a neuregulin 1 (NRG1) gene fusion with disease progression on or after prior systemic therapy. Bizengri is manufactured by Merus N.V.

• Bizengri is classified as a bispecific HER2- and HER3-directed antibody and is the first approved systemic therapy for patients with NSCLC or pancreatic cancer harboring an NRG1 gene fusion.
• The NRG1 gene encodes neuregulin, the ligand for HER3. NRG1 fusions are rare, occurring in less than 1% of NSCLC, pancreatic cancer and other solid tumors.
• Efficacy was evaluated in the eNRGy open-label study with 64 adults with NRG1 fusion-positive NSCLC and 30 adults with NRG1 fusion-positive pancreatic cancer with disease progression. In the NSCLC group, overall response rate (ORR) was 33% with a median duration of response (DOR) of 7.4 months. For pancreatic cancer, the ORR was 40% with a DOR ranging from 3.7 to 16.6 months.
• Bizengri is administered as an intravenous (IV) infusion over 4 hours, every 2 weeks until disease progression or unacceptable toxicity. Premedications should be administered before each infusion to reduce the risk of infusion-related reactions.
• The prescribing information includes a Boxed Warning for embryo-fetal toxicity. Warnings and precautions include infusion-related reactions, hypersensitivity and anaphylactic (allergic) reactions, interstitial lung disease (ILD)/pneumonitis (lung inflammation), and left ventricular dysfunction (weak heart that affects blood pumping).
• Common adverse reactions (≥ 10%) include diarrhea, musculoskeletal (muscle) pain, fatigue (tiredness), nausea, infusion-related reactions (IRR), dyspnea (shortness of breath), rash, constipation, vomiting, abdominal (stomach area) pain, and edema (fluid retention, swelling); as well as laboratory abnormalities.
• These indications are approved under accelerated approval based on overall response rate (ORR) and duration of response (DOR). Continued approval for these indications may be contingent upon verification and description of clinical benefit in confirmatory trials.

Nemluvio Gains New Use to Treat Patients with Moderate-to-Severe Atopic Dermatitis

On Dec. 13, 2024 the FDA cleared Galderma’s Nemluvio (nemolizumab) for its second indication: to treat patients 12 years and older with moderate-to-severe atopic dermatitis (eczema) with moderate-to-severe eczema (atopic dermatitis) in combination with prescription therapies used on the skin (topical) when the eczema is not well controlled by topical therapies alone. This approval follows Nemluvio’s approval for the treatment of adults with prurigo nodularis in August 2024.

• In atopic dermatitis, various proteins are involved in the skin inflammation. Nemluvio is the first monoclonal antibody that specifically targets and blocks IL-31 receptor alpha, a signaling protein (cytokine). IL-31 is produced by activated immune cells in the skin and specifically promotes itching and inflammation.
• Approval is based on positive results from the 16-week, phase 3, ARCADIA clinical trial program in 1,728 patients aged 12 years or older with moderate-to-severe atopic dermatitis. Results showed that patients treated with Nemluvio, injected subcutaneously (under the skin) every four weeks in combination with topical corticosteroids (TCS), with or without topical calcineurin inhibitors (TCI), achieved a significant 75% reduction in the Eczema Area and Severity Index (EASI) when compared to a group receiving placebo in combination with TCS (with or without TCI). Secondary endpoints like itch response in week 1 and effect on sleep disturbance were also met.
• It is available as a pre-filled pen for subcutaneous (under the skin) injection that requires reconstitution.
• Serious adverse events include serious allergic (hypersensitivity) reactions. Avoid use of live vaccines during treatment.
• The most common Nemluvio side effects are headache, atopic dermatitis, eczema, and nummular eczema (eczema presenting as scattered circular patches), which occur in ≥1% of patients in clinical trials.

FDA Approves Crenessity, a New Oral Treatment for Congenital Adrenal Hyperplasia

On Dec. 13, 2024, the FDA approved Crenessity (crinecerfont) capsules and oral solution from Neurocrine Biosciences. Crenessity is used in addition to glucocorticoid replacement to control androgens (a testosterone-like hormone) in patients 4 years of age and older with classic congenital adrenal hyperplasia (CAH), a rare genetic condition that affects the adrenal glands.

• People with classic CAH do not produce enough cortisol and produce too many androgens. Most cases of CAH are caused by variants of the CYP21A2 gene that lead to deficiency of the enzyme 21-hydroxylase (21-OH), resulting in a decrease in cortisol biosynthesis.
• Crinecerfont is a corticotropin-releasing factor (CRF) type 1 receptor antagonist that blocks the binding to inhibit adrenocorticotropic hormone (ACTH) secretion from the pituitary, thereby reducing ACTH-mediated adrenal androgen production. By reducing excessive adrenal androgen production, Crenessity reduces the amount of glucocorticoid treatment needed.
• Crenessity is administered orally (by mouth) twice daily. Approval was based on data from the CAHtalyst studies which showed a significant decrease in androstenedione (an androgen) levels compared to placebo (a pill with no medicine), which allowed significant glucocorticoid dose reductions.
• Warnings and precautions associated with Crenessity include hypersensitivity reactions, and the risk of acute adrenal insufficiency or adrenal crisis with inadequate concomitant glucocorticoid therapy.
• Warnings and precautions associated with Crenessity include hypersensitivity reactions, and the risk of acute adrenal insufficiency or adrenal crisis with inadequate concomitant glucocorticoid therapy.
• Common adverse reactions in adult patients include fatigue, headache, dizziness, and arthralgia (joint pain); Common adverse reactions in pediatric patients include headache, abdominal (stomach area) pain, fatigue, nasal congestion, and epistaxis (nose bleed).

FDA Clears Ensacove for ALK-Positive Locally Advanced or Metastatic Non-Small Cell Lung Cancer

Ensacove (ensartinib) is an oral anaplastic lymphoma kinase (ALK) inhibitor now FDA-approved for the treatment of adult patients with ALK-positive non-small cell lung cancer (NSCLC) in those who have not previously received an ALK-inhibitor.

• Ensartinib is a kinase inhibitor of anaplastic lymphoma kinase (ALK) and inhibits other kinases including MET and ROS1.
• The recommended dose of Ensacove is 225 mg orally once daily with or without food until disease progression or unacceptable toxicity. It is available as 25 and 100 mg capsules taken by mouth.
• Approval was based on the eXALT3 trial, open-label, randomized study in 290 patients with locally advanced or metastatic ALK-positive NSCLC who had not previously received an ALK-targeted therapy. Patients were randomized 1:1 to receive Ensacove or crizotinib (Xalkori). Ensacove significantly improved progression-free survival (PFS) compared to crizotinib (HR 0.56, p = 0.0007), with a median PFS of 25.8 months versus 12.7 months. There was no statistically significant difference in overall survival (OS) between the 2 treatments, a key secondary endpoint. Median OS was 63.2 months and 55.7 months in the Ensacove and crizotinib arms, respectively.
• Warnings include Interstitial Lung Disease (ILD) / Pneumonitis, liver toxicity, skin reactions, slowed heart rate, and high blood glucose (blood sugar), among others.
• Common adverse effects in at least 20% of patients included rash, musculoskeletal pain, constipation, cough, pruritus (itching), nausea, edema (fluid retention. swelling), pyrexia (fever), and fatigue.
• Ensacove is from Xcovery Holdings, Inc. and was FDA-approved on Dec. 18, 2024.

Ryoncil Now Approved as Mesenchymal Stromal Cell Therapy for Steroid-Refractory Acute Graft Versus Host Disease

The FDA has approved Ryoncil (remestemcel-L-rknd), an allogeneic bone marrow-derived mesenchymal stromal cell (MCS) therapy indicated for the treatment of steroid-refractory acute graft versus host disease (SR-aGvHD) in pediatric patients 2 months of age and older. Ryoncil is the first FDA-approved mesenchymal stromal cell (MSC) therapy.

• Steroid-refractory acute graft-versus-host disease is a serious and life-threatening condition that can occur as a complication of certain types of stem cell transplants. In allogeneic hematopoietic (blood) stem cell transplantation, a patient receives stem cells from a healthy donor to replace their own stem cells to help form new blood cells. This procedure may be done as for certain types of blood cancers or immune system disorders.
• The mechanism of action of Ryoncil may be related to its immunomodulatory effects. Data from in vitro studies demonstrate that mesenchymal stromal cell (MCS) therapy inhibits T cell activation as measured by proliferation and secretion of pro-inflammatory cytokines.
• Ryoncil’s effectiveness was based primarily on the rate and duration of response to treatment 28 days after initiating Ryoncil. In a single-arm, Phase 3 trial of children with SR-aGvHD, 70% achieved an overall response by Day 28, a measure that predicts survival in aGVHD. The full course was completed without interruption in more than 85% of patients.
• Ryoncil is contraindicated in patients with known hypersensitivity to dimethyl sulfoxide or porcine and bovine proteins. Patients should be premedicated with corticosteroids and antihistamines prior to infusion and monitored for hypersensitivity reactions during treatment with Ryoncil.
• The most common adverse reactions included infections, fever, hemorrhage, edema, abdominal (stomach area) pain and hypertension (high blood pressure). Complications such as hypersensitivity and acute infusion reactions, transmission of infectious disease or agents and ectopic tissue formation may occur following treatment with Ryoncil.
• Ryoncil is manufactured by Mesoblast, Inc. and was granted approval on Dec. 18, 2024.

FDA Approves Once-Monthly Tryngolza to Lower Triglycerides in Familial Chylomicronemia Syndrome

Tryngolza (olezarsen) is an APOC-III-directed antisense oligonucleotide now indicated to be used in addition to diet to lower triglycerides (a type of fat in the blood) in adults with familial chylomicronemia syndrome (FCS). It is an RNA-targeted medicine designed to lower the body's production of apoC-III, a protein that regulates triglyceride metabolism.

• Familial chylomicronemia syndrome (FCS) is a rare, genetic form of severe hypertriglyceridemia (sHTG) that can lead to potentially life-threatening acute pancreatitis (AP). Patients often have triglyceride levels of more than 880 mg/dL.
• Tryngolza is the first treatment that significantly reduces triglyceride levels in adults with FCS and provides reduction in acute pancreatitis (AP) events when used with an appropriate diet (equal to or less than 20 grams of fat per day). It is self-administered via an auto-injector once monthly.
• Approval was based on the Phase 3, placebo-controlled BALANCE studies in 66 adults where Tryngolza 80 mg demonstrated a statistically significant mean reduction in triglyceride levels of 42.5% from baseline to 6 months (p=0.0084), 57% at 12 months and clinically meaningful reduction in AP events over 12 months (1 AP episode in Tryngolza group vs. 11 in placebo).
• Warnings include hypersensitivity (allergic) reactions. The most common adverse reactions in at least 5% of Tryngolza-treated patients were injection site reactions, decreased platelet count (a blood cell that helps with clotting) and arthralgia (joint pain).
• Tryngolza, approved on Dec. 19, 2024, is manufactured by Ionis Pharmaceuticals, Inc. and is expected to be commercially available at the end of 2024.

FDA Clears Alyftrek, a Triple Combination Therapy for Cystic Fibrosis

On Dec. 20, 2024 the FDA cleared Vertex Pharmaceutical’s Alyftrek (vanzacaftor, tezacaftor and deutivacaftor) indicated for the treatment of cystic fibrosis (CF) in patients aged 6 years and older who have at least one F508del mutation or other mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to this treatment. Alyftrek is the first once-daily CFTR modulator.

• Cystic fibrosis (CF) is a rare genetic disease caused by mutations in the CFTR gene, which leads to decreased quantity and/or function of the CFTR protein channel at the cell surface. This can cause a poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus, chronic lung infections and progressive lung damage that eventually leads to death for many patients.
• Vanzacaftor and tezacaftor are designed to increase the amount of CFTR protein at the cell surface. Deutivacaftor helps to keep CFTR proteins at the cell surface open longer to improve the flow of salt and water across the cell membrane, which helps hydrate and clear mucus from the airways.
• Approval was based on Phase 3 studies in people with CF 12 years of age and older. The primary endpoint was met when compared to Trikafta - non-inferiority on absolute change from baseline in percent predicted forced expiratory volume (ppFEV1), which measures how well a person’s lungs work. All key secondary endpoints were also met. In the Phase 3 study of children with CF ages 6 to 11 years safety endpoints, the primary outcome in this age group, were also met.
• Alyftrek carries a Boxed Warning for drug-induced liver injury and liver failure. Warnings and precautions also include hypersensitivity (allergic) reactions, including cases of anaphylaxis, reduced effectiveness of Alyftrek with use of strong or moderate CYP3A inducers, and increased risk of cataracts.
• Serious adverse reactions include influenza (flu), elevated liver enzymes, depression and fainting. Common side effects include cough, common cold, upper respiratory tract infection, headache, and mouth / throat pain, among others.
• Tezacaftor is contained in the approved combination CF treatments Symdeko (ivacaftor/tezacaftor and ivacaftor) and Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor).

Posted December 2024

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