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Monthly News Roundup - January 2025

Medically reviewed by Leigh Ann Anderson, PharmD. Last updated on Jan 31, 2025.

FDA Clears Oral Journavx, a First-in-Class, Non-Opioid Pain Treatment from Vertex Pharmaceuticals

On January 30th the FDA approved Journavx (suzetrigine) 50 milligram oral tablets, a first-in-class, non-opioid analgesic to treat moderate to severe acute pain in adults. Journavx is a sodium channel blocker that relieves pain by blocking pain-sensing nerves in the body from transmitting pain messages to the spinal cord and brain. It is the first new class of pain medicine approved in more than 2 decades, and may offer an alternative to opioid treatments like oxycodone or hydrocodone.

• Acute pain is common and is often due to trauma, surgery or other medical issues. Journavx works by selectively blocking NaV1.8, a sodium channel on pain-sensing nerve cells (neurons) which inhibits pain signals to the spinal cord and brain. It is not expected to have addictive potential like opioids because it works in the peripheral nervous system - not the brain.
• Developing new, non-opioid pain treatment options has been a long-time goal of the FDA. This application received Breakthrough Therapy, Fast Track and Priority Review status by the FDA.
• Approval was based on 2 randomized trials of acute surgical pain following abdominoplasty (“tummy tuck”) and bunionectomy (removal of bunion), with over 2,100 participants.. Eligible patients were randomized to receive the following for 48 hours: oral Journavx at an initial loading dose of 100 mg, followed by 50 mg every 12 hours; placebo; or hydrocodone bitartrate/acetaminophen (HB/APAP), 5 mg/325 mg every 6 hours. All participants in the trials with inadequate pain control were permitted to use 400 mg ibuprofen every 6 hours as needed for “rescue” pain medication.
• Efficacy was evaluated by the time-weighted sum of the pain intensity difference from 0 to 48 hours (SPID48), a common endpoint in acute pain trials.
• Both trials demonstrated a statistically significant superior reduction in pain with Journavx compared to placebo.
• The least squares (LS) mean difference vs. placebo in the SPID48 was 48.4 (95% CI: 33.6, 63.1; P<0.0001) with abdominoplasty and 29.3 (95% CI: 14.0, 44.6; P=0.0002) after bunion removal.
• The median time to meaningful pain relief was reported as 2 hours for abdominoplasty and 4 hours for bunion removal compared with 8 hours for placebo. Perceptible pain relief began within 30 to 60 minutes.
• Based on SPID48, neither study showed that Journavx was better at treating pain when compared to HB/APAP.
• The recommended starting dose of Journavx is 100 mg orally on an empty stomach at least 1 hour before or 2 hours after food to avoid delay in onset of action. Clear liquids may be consumed during this time (e.g., water, apple juice, vegetable broth, tea, black coffee). Starting 12 hours after the initial dose, the recommended dose is 50 mg orally every 12 hours, with or without food. Journavx is available as a 50 mg oral tablet. Treatment beyond 14 days has not been studied.
• Do not use Journavx with strong CYP3A inhibitors (contraindicated) or in patients with severe liver impairment. Avoid food or drink containing grapefruit during treatment. Reduced doses are recommended in patients taking moderate CYP3A inhibitors and those with moderate liver impairment. Avoid use in patients with eGFR <15 mL/min (kidney impairment).
• Warnings and precautions include moderate and severe liver impairment. In studies, the most common adverse reactions (in at least 1% of patients and greater than placebo) were itching (2.1%), muscle spasms (1.3%), increased blood level of creatine phosphokinase (1.1%) and rash (1.1%). Nausea and vomiting was also reported in up to 20% of patients, but was less than HB/APAP (active control) or placebo groups.
• Vertex has established a wholesale acquisition cost for Journavx at $15.50 per pill. Vertex is also studying Journavx in other painful conditions, such as diabetic peripheral neuropathy and lower back disc inflammation, “pinched nerve” and pain (lumbosacral radiculopathy).

FDA Approves Ozempic as First GLP-1 Agonist to Reduce Chronic Kidney Disease

Novo Nordisk has announced the FDA approval of Ozempic (semaglutide) to reduce the risk of kidney disease worsening (eGFR decline), kidney failure (end-stage kidney disease), and death due to cardiovascular disease in adults with type 2 diabetes (T2D) and chronic kidney disease (CKD). It is the first glucagon-like peptide-1 (GLP-1) agonist to receive this indication.

• Ozempic was first approved in 2017 to treat adults with T2D to improve glycemic (blood sugar) control, along with diet and exercise; then again in 2020 to reduce the risk of major cardiovascular events (such as heart attack, stroke, or death) in adults also with known heart disease.
• CKD is a common complication of T2D, occurring in about 40% of people with T2D and can eventually lead to the need for dialysis. CKD is estimated to affect about 37 million adults in the U.S.
• Approval was based on the Phase 3b FLOW trial, a randomized, double-blind, placebo-controlled trial with a median follow up of 3.4 years in 3,533 adults with T2D and CKD. Ozempic 1 mg given subcutaneously (under the skin) once-weekly was compared to placebo. The primary endpoint was met, with Ozempic demonstrating a 24% relative risk reduction (hazard ratio, 0.76; 95% confidence interval [CI], 0.66 to 0.88; P=0.0003) of kidney disease worsening, kidney failure, and death due to cardiovascular disease (4.9% absolute risk reduction at 3 years) compared to placebo, when added to standard of care. A secondary outcome, ​​the risk of death from any cause was reduced by 20%.
• After an initial dose titration over 8 weeks to lessen GI symptoms, the final maintenance dose is 1 mg given subcutaneously once weekly.
• Ozempic carries a Boxed Warning for risk of thyroid C-cell tumors and should not be used in patients with a personal or family history of medullary thyroid cancer MTC or in patients with Multiple Endocrine Neoplasia syndrome type 2 (MEN 2). Other warnings include pancreatitis, diabetic retinopathy, hypoglycemia (low blood sugar), sudden kidney injury and serious allergic reactions, among others.
• In the study, serious adverse events were reported in fewer participants in the Ozempic group than placebo (49.6% vs. 53.8%). In general, the most common adverse effects in at least 5 % of people include stomach side effects such as nausea, vomiting, diarrhea, abdominal (stomach) pain and constipation.

Enhertu Use Expanded in HER2-Low or HER2-Ultralow Metastatic Breast Cancer

The FDA has expanded the indications of Enhertu (fam-trastuzumab deruxtecan-nxki) for the treatment of adults with hormone receptor (HR)-positive, HER2-low (IHC 1+ or IHC 2+/ISH-) or HER2-ultralow (IHC 0 with membrane staining) breast cancer, that cannot be removed by surgery or has spread in the body, and that has progressed on one or more endocrine therapies in the metastatic setting. Eligible patients are determined by an FDA-approved test.

• In some breast cancer cells, the human epidermal growth factor receptor 2 (HER2) protein is found on the cell surface and can cause cells to grow too quickly and spread. In some people with breast cancer identified as HER2-negative, low and ultra-low levels of the HER2 protein may still exist. These cells contain fewer HER2 proteins than do HER2-positive cancer cells, but may be amenable to treatment.
• Enhertu is a chemotherapy and targeted drug combination known as a HER2-directed antibody-drug conjugate (ADC). It combines two types of cancer medicines to help lengthen the time before the cancer progresses and to improve patient survival.
• Approval for this use was based on the results from the DESTINY-Breast06 Phase III trial. Enhertu showed a 36% reduction in the risk of disease progression or death versus chemotherapy (hazard ratio [HR] 0.64; 95% confidence interval [CI]: 0.54-0.76; p<0.0001) in the overall trial population of patients with chemotherapy-naïve HER2-low or HER2-ultralow metastatic breast cancer.
• A median progression-free survival (PFS) of 13.2 months was seen in patients randomised to Enhertu compared to 8.1 months in those randomised to chemotherapy. The confirmed objective response rate (ORR) in the overall trial population was 62.6% for Enhertu versus 34.4% for chemotherapy.
• FDA labeling for Enhertu contains a Boxed Warning for interstitial lung disease and embryo-fetal toxicity. Common adverse reactions in at least 20% of patients include: decreased red and white blood cell counts, nausea, tiredness, decreased platelet count, increased liver enzymes, vomiting, hair loss, constipation, decreased blood potassium, decreased appetite, diarrhea, and muscle and bone pain.
• Enhertu, a collaboration between AstraZeneca and Daiichi Sankyo, is also approved to treat certain types of stomach cancer, non-small cell lung cancer and solid tumors that have HER2 gene mutations.

Datroway Cleared for Patients with Metastatic, HR-Positive, HER2-Negative Breast Cancer

The FDA has cleared Datroway (datopotamab deruxtecan-dlnk), a TROP2-directed antibody and topoisomerase inhibitor conjugate used for the treatment of adults with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative (IHC 0, IHC1+ or IHC2+/ISH-) breast cancer that cannot be removed with surgery or has spread in the body, and who have received prior endocrine-based therapy and chemotherapy.

• Datroway is an antibody-drug conjugate (ADC) composed of a humanized anti-TROP2 IgG1 monoclonal antibody (mAb), covalently linked to a cytotoxic topoisomerase I inhibitor, via a tetrapeptide-based cleavable linker. Datroway works by binding to TROP2 (trophoblast cell surface antigen 2) on TROP2 expressing tumors where it undergoes internalization into cancer cells leading to DNA damage and cell death.
• Datroway is given as an intravenous (IV) infusion, once every 3 weeks (21-day cycle), until disease progression or unacceptable toxicity.
• Approval was based on results from the TROPION-Breast01 Phase III trial, an open-label trial with 732 patients who were randomized to receive Datroway or investigator’s choice of chemotherapy (eribulin, capecitabine, vinorelbine or gemcitabine). The primary outcome measures were progression-free survival (PFS) and overall survival (OS).
• Progression-free survival (PFS) is the length of time during and after the treatment of the cancer that a patient lives with the disease but it does not get worse. In this study, median PFS was found to be significant at 6.9 months in the Datroway arm and 4.9 months in the chemotherapy arm (p-value <0.0001). Datroway significantly reduced the risk of disease progression or death by 37% compared to the investigator's choice of chemotherapy.
• Overall survival (OS) is the length of time from the start of treatment that a patient diagnosed with the cancer is still alive. In this study, the median OS was not found to be statistically significant - 18.6 months in the Datroway arm and 18.3 months in the chemotherapy arm. A secondary endpoint, the median duration of response (DOR) was 6.7 months in the Datroway group and 5.7 months in the chemotherapy arms.
• Warnings and precautions include: interstitial lung disease (ILD) and pneumonitis (which can be fatal), ocular (eye) adverse reactions (including dry eye, keratitis, blepharitis and meibomian gland dysfunction, increased lacrimation, conjunctivitis, and blurred vision), stomatitis and oral mucositis (mouth ulcers / inflammation), and fetal harm.
• Some of the most common adverse reactions (in at least 20% of patients), including laboratory abnormalities, were stomatitis, nausea, fatigue, decreased calcium, alopecia (hair loss), decreased white blood cells, decreased hemoglobin, constipation, dry eye and increased liver enzymes, among others.
• Datroway was approved on Jan. 17, 2025 and is made by Daiichi Sankyo and AstraZeneca.

Calquence Now Approved for Patients with Previously Untreated Mantle Cell Lymphoma

The FDA has approved oral Calquence (acalabrutinib) to be used combination with cancer agents bendamustine and rituximab for the treatment of adult patients with previously untreated mantle cell lymphoma (MCL) who are ineligible for autologous hematopoietic stem cell transplantation (HSCT). This approval converts the accelerated approval from 2017 to a full approval for this use.

• Calquence is also indicated to treat adults with MCL who have received at least one prior therapy, and adults with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).
• Mantle cell lymphoma is a rare blood cancer and a form of non-Hodgkin's lymphoma that develops from mature B lymphocytes in a region of the lymph nodes called the mantle zone. Symptoms can include swollen lymph glands, fever, night sweats, fatigue, unexplained weight loss, and abdominal swelling. MCL makes up roughly 3% to 6% of non-Hodgkin lymphomas.
• Calquence is a highly selective, targeted Bruton tyrosine kinase (BTK) inhibitor and works by blocking this protein to keep cancer cells from growing. Calquence is not classified as a chemotherapeutic agent.
• Approval was based on the pivotal ECHO trial with 598 patients who were ≥ 65 years of age that showed Calquence plus bendamustine and rituximab (BR) reduced the risk of disease progression or death by 27% compared to placebo plus BR (hazard ratio [HR] 0.73; 95% confidence interval [CI] 0.57-0.94; p=0.016). Median progression-free survival (PFS), the primary outcome, was 66.4 months for patients treated with the Calquence combination vs 49.6 months with chemoimmunotherapy alone (a difference of 16.8 months).
• Calquence capsules or tablets are taken by mouth twice daily, swallowed whole, with or without food. Unlike the capsule, the tablet formulation can be co-administered with gastric acid-reducing agents, such as proton pump inhibitors (PPIs), antacids and H2-receptor antagonists (H2RAs).
• The most common adverse reactions include: diarrhea, upper respiratory tract infection, headache, musculoskeletal pain, lower respiratory tract infection, and fatigue, in addition to reductions in certain hematology lab values (neutrophils, lymphocytes, platelets, hemoglobin).
• This new indication was approved on Jan. 16, 2025. Calquence is manufactured by AstraZeneca.

Lilly's Omvoh Gains New Indication for Crohn's Disease in Adults

This past month the FDA cleared Omvoh (mirikizumab-mrkz) for the treatment of moderate to severe active Crohn's disease in adults. Previously, Omvoh was also approved for the treatment of moderate to severe active ulcerative colitis in adults.

• Crohn’s disease is a long-term (chronic) inflammatory bowel disease with disruptive stomach symptoms such as abdominal pain, frequent bowel movements and bowel urgency. Crohn's disease typically begins between ages 15 and 40.
• Interleukin-23 (IL-23) is a naturally occurring cytokine involved in inflammatory and immune responses. Omvoh works as an IL-23 antagonist and targets the p19 subunit of IL-23. By blocking IL-23, Omvoh inhibits the release of cytokines and chemokines, proteins that can cause gastrointestinal inflammation.
• Approval was based on the Phase 3 VIVID-1 placebo-controlled study in adults with moderately to severely active Crohn's disease who had an inadequate response or intolerance to corticosteroids, immunomodulators (azathioprine, 6-mercaptopurine and methotrexate), and/or biologics (TNF blockers, integrin receptor antagonists).
• At one year, 53% of patients treated with Omvoh achieved clinical remission (assessed by Crohn's Disease Activity Index) compared to 36% on placebo. In addition, 46% of patients treated with Omvoh had an endoscopic response (visible healing of the intestinal lining) compared to 23% on placebo.
• Both primary endpoints were met with statistical significance (p<0.001) at one year: one-year clinical remission by Crohn's Disease Activity Index (53% Omvoh vs 36% placebo); and endoscopic response with visible healing of the intestinal lining (46% Omvoh vs 23% placebo). One-third of patients on Omvoh achieved an early improvement in endoscopic response at 3 months (vs 11% on placebo).
• In an ongoing open-label study in patients who achieved clinical remission and endoscopic response at one year in VIVID-1, nearly 90% of patients maintained clinical remission with one year of additional treatment (two years of continuous treatment).
• Omvoh is given for the first 3 doses as an intravenous (IV) infusion every 4 weeks by a healthcare provider; then as an ongoing subcutaneous (under the skin) maintenance dose at week 12 and thereafter. Patients may self-inject the subcutaneous form of Omvoh after training from a healthcare provider.
• Warnings and precautions include hypersensitivity (allergic) reactions, risk of infection, tuberculosis (TB), and liver toxicity. Avoid use of live vaccines with Omvoh. Do not administer Omvoh to patients with active TB infection.
• Common side effects in Crohn’s disease patients include upper respiratory tract infections, injection site reactions, headache, joint / muscle pain and elevated liver tests.
• Omvoh was approved for Crohn’s disease on Jan. 15, 2025 and is manufactured by Eli Lilly and Company.

FDA Approves Leqembi for Monthly Maintenance Dosing in Early Alzheimer’s Disease

On January 27 the FDA approved Leqembi (lecanemab-irmb) injection for once every 4 weeks intravenous (IV) maintenance dosing for the treatment of Alzheimer's disease in patients with mild cognitive impairment or mild dementia stage of disease (early disease).

• The new dosing routine may offer more convenience after 1.5 years. Patients initially must complete Leqemvi at a dose of once every two weeks for 18 months, then they may be able to transition to the once a month dosing schedule (or they can remain on the every 2 week dosing schedule).
• Leqembi is an amyloid beta-directed antibody indicated for the treatment of Alzheimer’s disease. Treatment should be initiated in patients with mild cognitive impairment or mild dementia stage of disease, the population in which treatment was initiated in clinical trials.
• Leqembi has been shown to reduce brain amyloid and modestly slow cognitive decline in patients with early Alzheimer's disease. It is thought to slow down the progression of Alzheimer’s by neutralizing and eliminating the most toxic amyloid-beta aggregates.
• Approval for the once per month dosing schedule was based on modeling of observed data (Study 201, 301 [Clarity AD study] and long-term extension data) that predicted that transitioning to once every 4 weeks maintenance dosing after 18 months of once every 2 weeks treatment will maintain clinical and biomarker benefits of therapy.
• Leqembi labeling contains a Boxed Warning for Amyloid Related Imaging Abnormalities (ARIA), side effects seen with MRI that are usually asymptomatic and resolve with time, but can be serious in some patients. ARIA-E (Edema) is a temporary swelling in the brain and ARIA-H (Hemosiderin) leads to small areas of bleeding and iron deposits in the brain.
• The risk of ARIA was greater in patients who are ApoE4 homozygotes compared to heterozygotes and noncarriers. Clinicians should complete testing for ApoE4 status prior to initiating treatment.
• Serious intracerebral hemorrhage greater than 1 cm have occurred in patients treated with this class of medications. The use of anticoagulant or thrombolytic medications while taking Leqembi may increase the risk of bleeding in the brain. Enhanced clinical vigilance for ARIA is recommended during the first 14 weeks of treatment with Leqembi.
• The most common adverse reactions include infusion-related reactions, amyloid related imaging abnormality-microhemorrhages, amyloid related imaging abnormalityedema/effusion, and headache. If a patient experiences symptoms suggestive of ARIA, clinical evaluation should be performed, including MRI scanning if indicated.
• Leqembi is from Eisai Inc. and Biogen and was approved for this indication on Jan. 27, 2025.

Spravato Nasal Spray Approved as Single Therapy for Treatment-Resistant Depression

The FDA has approved Spravato (esketamine) nasal spray to be used alone (as monotherapy) for adults with treatment-resistant depression (TRD). It is also approved for TRD in adults in conjunction with an oral antidepressant, as well as for depressive symptoms in adults with major depressive disorder (MDD) with acute suicidal ideation or behavior in conjunction with an oral antidepressant

• Spravato is classified as a non-competitive N-methyl D-aspartate (NMDA) receptor antagonist and works by targeting the excitatory neurotransmitter glutamate in the brain, although the exact mechanism in depression is not known.
• Spravato contains esketamine, which is the s-enantiomer of ketamine. It is classified as a CIII controlled substance. Because of the risk of central nervous system side effects and abuse, it is used only in a healthcare setting through a restricted distribution system (REMS). Patients cannot take the drug home, but will self-administer nasal Spravato at a certified doctor's office. Patients need to arrange transportation home following treatment.
• For TRD, initial doses of the nasal spray are administered twice per week during the Induction Phase (Weeks 1 to 4), then once weekly in the Maintenance Phase (Weeks 5 to 8). From Week 9 and onwards, doses are given once a week or every 2 weeks, based on individual results, utilizing the least frequent dosing to maintain a response. Assess blood pressure prior to and after administration. Each device delivers two sprays containing a total of 28 mg of esketamine.
• Approval was based on positive results from the randomized, double-blind, multicenter, placebo-controlled study in which Spravato used alone showed a rapid and superior improvement in Montgomery-Asberg Depression Rating Scale (MADRS) total score vs. placebo. At week 4, 7.6% of patients taking placebo and 22.5% of patients taking Spravato achieved remission (MADRS total score ≤ 12).
• No new safety concerns have been identified for this use. Spravato labeling carries a Boxed Warning for risk of sedation, dissociation, respiratory depression, abuse and misuse, and suicidal thoughts and behaviors.
• The most common side effects (incidence 5%) include dissociation (feeling detached from one’s surroundings), dizziness, nausea, sedation, vertigo (spinning sensation), decreased feeling or sensitivity (hypoesthesia), anxiety, lethargy, increased blood pressure, vomiting, feeling drunk, headache and euphoric (very happy or excited) mood.
• Spravato is from Janssen Pharmaceuticals and was approved for this use on Jan. 21, 2025.

Lumakras + Vectibix Gain FDA Clearance to Treat Colorectal Cancer

The FDA has approved Lumakras (sotorasib) in combination with Vectibix (panitumumab) for the treatment of adult patients with KRAS G12C-mutated metastatic colorectal cancer (mCRC), as determined by an FDA-approved test, who have received prior fluoropyrimidine-, oxaliplatin- and irinotecan-based chemotherapy. Lumakras was also approved in 2021 to treat certain adults with non-small cell lung cancer (NSCLC) with an abnormal KRAS G12C gene.

• Lumakras is a targeted, biomarker-driven combination therapy. KRAS is a protein that relays messages to cells to begin or end cell growth. Lumakras is a KRAS G12C inhibitor (blocker) and works by targeting cancers with the mutated KRAS G12C gene to help stop the cancer from growing.
• Approval was based on the phase III, randomized, open-label CodeBreaK 300 study which compared Lumakras at two different doses (960 mg daily or 240 mg daily) in combination with Vectibix to the investigator's choice of standard-of-care (trifluridine and tipiracil or regorafenib) in patients with chemorefractory KRAS G12C-mutated mCRC.
• Lumakras plus Vectibix is the first targeted treatment combination for chemorefractory KRAS G12C-mutated mCRC to show superior progression-free survival (PFS) compared to the investigated standard-of-care. Study results showed that Lumakras 960 mg daily plus Vectibix (n=53) showed an improved median PFS of 5.6 months (4.2, 6.3) compared to 2 months (1.9, 3.9) on investigator's choice of care (n=54); (p=0.005). PFS of Lumakras 240 mg daily plus Vectibix (n=53) compared to the investigator's choice was not statistically significant.
• The recommended dose of Lumakras is 960 mg by mouth once daily, and the recommended dose of Vectibix is 6 mg/kg intravenously (into a vein) every 2 weeks. Follow your prescriber’s instructions exactly.
• The most common adverse reactions (in at least 20% of people) are rash (87%), dry skin (28%), diarrhea (28%), stomatitis / mouth sores and inflammation (26%), fatigue (21%) and muscle / joint pain (21%).
• Lumakras, from Amgen, was approved for the new mCRC indication on Jan. 16, 2025.

FDA Approves Grafapex Preparative Regimen for Allogeneic Hematopoietic Stem Cell Transplantation

In January, the FDA cleared Grafapex (treosulfan), an alkylating agent used with fludarabine as a preparative regimen for allogeneic hematopoietic stem cell transplantation (alloHSCT) in adults and children one year of age and older with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS).

• AlloHSCT is a procedure that involves the use of healthy stem cells from a donor to replace stem cells for a patient. A conditioning treatment is given to the patient before receiving alloHSCT to destroy any remaining cancer cells and weaken the immune system so that the donor cells are not rejected. An allogeneic stem cell transplant is most often used to treat blood cancers, such as leukemia and lymphoma, and certain types of blood or immune system disorders.
• Grafapex is a DNA alkylating agent that works by interfering with DNA replication and RNA transcription, and disrupting nucleic acid function to cause cell death. It is administered as a 2 hour intravenous (IV) infusion, given on 3 consecutive days in conjunction with fludarabine before hematopoietic stem cell infusion.
• FDA approval was supported by data from the MC-FludT.14/L Trial II which compared Grafapex plus fludarabine (n=280) to busulfan plus fludarabine (n=290) as a preparative regimen for alloHCT. Results showed that the Grafapex / fludarabine regimen (compared to the busulfan / fludarabine regimen) led to a:
• 33% reduction in the risk of death in the overall population (HR, 0.67; 95% CI, 0.51-0.90)
• 27% reduction in patients with AML (HR, 0.73; 95% CI, 0.51-1.06)
• 36% reduction in patients with MDS (HR, 0.64; 95% CI, 0.40-1.02)
• Warnings and precautions include seizures, skin disorders, injection site reactions and local tissue necrosis (tissue death), increased risk of a secondary malignancy (cancer), embryo-fetal toxicity.
• Common adverse reactions (in at least 20% of patients in studies) include muscle / bone pain, mouth sores / inflammation, fever, nausea, edema (fluid retention, swelling), infection, laboratory abnormalities and vomiting.
• Grafapex is designated as an Orphan Drug and will have up to 7.5 years of regulatory exclusivity for this indication. It’s expected to be commercially available in the first half of this year. It is manufactured by Medexus Pharmaceuticals and was approved on Jan. 22, 2025.

Posted January 2025

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