Monthly News Roundup - January 2024

Medically reviewed by Leigh Ann Anderson, PharmD. Last updated on Jan 30, 2024.

First-in-Class Zelsuvmi Topical Gel Cleared for Molluscum Contagiosum

In January, the FDA cleared Zelsuvmi (berdazimer sodium 10.3%), a topical treatment for molluscum contagiosum (MC) in adults and children 1 year of age and older. Molluscum contagiosum is a common, contagious viral skin infection characterized by small, raised, skin-colored to red lesions on the body. It can cause inflammation, itching, and bacterial infection. Approximately 6 million Americans, primarily children, are infected each year, but up to 73% of children go untreated.

It is thought Zelsuvmi works by releasing nitric oxide which affects NF-κB, immunomodulation, inflammation, cytokine production, and apoptosis likely through S-nitrosylation of proteins. Nitric oxide also has cytotoxic functions that affect viral replication.
Approval was based on the results of the B-SIMPLE Phase 3 trials which demonstrated a reduction in lesion counts and also showed that the treatment was well tolerated when used once a day. In the B-SIMPLE4 study with 891 participants, results at week 12 showed a complete clearance of MC lesions in 32.4% of patients randomized to Zelsuvmi compared to 19.7% assigned to the inactive vehicle gel.
Zelsuvmi, an at-home treatment, is applied once daily to each molluscum contagiosum lesion for up to 12 weeks. It is supplied as two tubes containing berdazimer gel and hydrogel, which are mixed together immediately prior to application.
Warnings and precautions associated with Zelsuvmi are application site reactions, including allergic contact dermatitis.
Common side effects are: application site reactions include pain, burning or stinging sensations (18.7%), redness (11.7%), itching (5.7%), exfoliation (5%), dermatitis (4.9%), and swelling (3.5%), among others.
Zelsuvmi is manufactured by Ligand Pharmaceuticals and is expected to be available in the second half of 2024.

Casgevy Lands Second Approval for Transfusion-Dependent Beta Thalassemia

This past month the FDA approved Casgevy (exagamglogene autotemcel), a CRISPR / Cas9 genome-edited, one time cell therapy for the treatment of transfusion-dependent beta-thalassemia in patients 12 years and older. This approval came on the heels of a landmark approval last month, with Casgevy as the first CRISPR / Cas9 genome-edited cell therapy to treat sickle cell disease (SCD) in patients 12 years and older with frequent vaso-occlusive crises (VOCs).

Beta-thalassemia is a life-threatening, inherited blood disorder with the transfusion-dependent type being the most severe. Beta thalassemia is caused by changes in the hemoglobin beta (HBB) gene. Patients usually require lifelong blood transfusions and iron chelation therapy.
Casgevy, developed by Vertex Pharmaceuticals and CRISPR Therapeutics, is made specifically for each patient by editing the BCL11A gene and no donor is needed. CRISPR / Cas9 can be directed to cut DNA in targeted areas, enabling the ability to accurately edit DNA where it was cut.
Casgevy works by increasing the production of hemoglobin F (fetal hemoglobin or HbF) which can improve the production and function of red blood cells. This can eliminate the need for regular blood transfusions in people with beta-thalassemia.
In an ongoing, open-label, study with 35 evaluable patients, researchers evaluated the proportion of patients achieving transfusion independence for 12 consecutive months (TI12). The TI12 responder rate was 32 / 35 patients. All patients who achieved TI12 remained transfusion-independent, with a median duration of transfusion-independence of 20.8 months.
The most common side effects (≥ 10%) in patients with TDT included febrile neutropenia (fever and low white blood cells), mucositis (mouth / anal sores), veno-occlusive liver disease, decreased appetite, epistaxis (nosebleed), and infusion-related reactions. Laboratory adverse reactions were common and included neutropenia, thrombocytopenia, leukopenia and anemia.

Merck’s Keytruda Okayed for Third Cervical Cancer Indication

In January, the FDA approved Keytruda (pembrolizumab), an anti-PD-1 therapy, in combination with chemoradiotherapy (CRT) for the treatment of patients with FIGO (International Federation of Gynecology and Obstetrics) 2014 Stage III-IVA cervical cancer. Cervical cancer forms in the cells lining the cervix, which is the lower part of the uterus, and is the fourth most common cancer in women globally.

Keytruda is the first anti-PD-1 therapy approved in combination with chemoradiotherapy for FIGO 2014 Stage III-IVA cervical cancer, regardless of PD-L1 expression.
Approval is based on results from the Phase 3 KEYNOTE-A18 trial. The median duration of exposure to Keytruda was 12.1 months (range, 1 day to 27 months). Keytruda plus CRT demonstrated an improvement in progression-free survival (PFS), reducing the risk of disease progression or death by 41% compared to placebo plus CRT in patients with FIGO 2014 Stage III-IVA disease. Median PFS was not reached in either group. 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.
The dose for cervical cancer is 200 mg every 3 weeks or 400 mg every 6 weeks given as an intravenous (IV) infusion. Treatment is continued until disease progression, unacceptable toxicity, or for up to 24 months for Keytruda.
Serious adverse reactions occurring in ≥1% of patients included urinary tract infection (2.7%), urosepsis (1.4%), and sepsis (1%). Common side effects (≥10%) included: nausea, diarrhea, vomiting, urinary tract infection, fatigue, hypothyroidism, and laboratory abnormalities, among others.
This marks the third FDA-approved Keytruda use in cervical cancer and its 39th indication overall.

Dupixent Label Expanded for Atopic Eczema and Eosinophilic Esophagitis Populations

In January the FDA approved updated labeling for Regeneron’s Dupixent (dupilumab), an interleukin-4 receptor alpha antagonist, for its uses in atopic dermatitis and eosinophilic esophagitis (EoE).

In the Phase 3 LIBERTY-AD-HAFT placebo-controlled trial, the safety and efficacy of Dupixent was evaluated in 133 adult and adolescent (aged 12 to 17 years) patients with atopic dermatitis (eczema) with moderate-to-severe hand and / or foot involvement and a poor response to topical corticosteroids. At 16 weeks, patients treated with Dupixent experienced the following:

40% achieved clear or almost clear skin on hands and feet compared to 17% with placebo, the primary endpoint (a score of 0 or 1 on the Investigator Global Assessment Scale).
52% saw a clinically meaningful reduction in itch on hands and feet compared to 14% with placebo, a key secondary endpoint.
Dupixent is approved for the treatment of adult and pediatric patients aged 6 months and older with moderate-to-severe AD whose disease is not adequately controlled.

Later in the month the FDA also cleared Dupixent for the treatment of pediatric patients aged 1 to 11 years, weighing at least 15 kg (33 lb), with eosinophilic esophagitis (EoE). Dupixent is the only medicine currently approved in the U.S. to treat this patient population.

This new use expanded the May 2022 EoE approval for patients aged 12 years and older, weighing at least 40 kg (88 lb).
Approval was based on the Phase 3 EoE KIDS trial evaluating the efficacy and safety of Dupixent in children aged 1 to 11 years with EoE. At 16 weeks, 66% of children who received higher dose Dupixent at tiered dosing regimens based on weight (n=32) achieved histological disease remission (≤6 eosinophils/high power field), the primary endpoint, compared to 3% for placebo (n=29).
Histological remission was sustained at week 52 in 53% of children treated with Dupixent or switched to Dupixent from placebo.

Dupixent is also approved by the FDA to treat asthma, chronic rhinosinusitis with nasal polyposis, and prurigo nodularis.

Janssen’s Balversa for Metastatic Bladder Cancer Gains Full FDA Approval

In April 2019 the FDA first granted accelerated approval for Balversa (erdafitinib), a once-daily oral FGFR kinase inhibitor to treat adult patients with locally advanced or metastatic bladder (urothelial) cancer. Now, this past month, the FDA has cleared Balserva’s full approval based on the clinical and overall survival benefit observed in the Phase 3 THOR study.

Balversa is approved for the treatment of adult patients with locally advanced or metastatic urothelial carcinoma (mUC) with susceptible FGFR3 genetic alterations whose disease has progressed on or after at least one line of prior systemic therapy. Balversa is not recommended for patients who are eligible for and have not received prior PD-1 or PD-L1 inhibitor therapy.
Urothelial cancer is the most common form of bladder cancer, representing more than 90% of all bladder cancers. Approximately 20% of patients with mUC have FGFR3 genetic alterations.
Studies showed a 36% reduction in the risk of death with Balversa when compared to chemotherapy in patients previously treated with a PD-1 or PD-L1 inhibitor. Those in the Balversa arm survived a median of over 4 months longer.
The recommended initial dosage is 8 mg orally once daily, with a dose increase to 9 mg daily if criteria are met. Swallow the tablet whole with or without food.
Serious reactions in >2% of patients included urinary tract infection (4.4%), hematuria (3.7%), hyponatremia (2.2%), and acute kidney injury (2.2%). The most common side effects include nail disorders (70%), diarrhea (63%), stomatitis / mouth inflammation (56%), taste changes (30%), and laboratory changes, among others.

FDA Approves Takeda’s Hyqvia to Treat Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)

Hyqvia (immune globulin and hyaluronidase) is a subcutaneous immune globulin combination now approved as maintenance therapy to prevent neuromuscular disability relapse in chronic inflammatory demyelinating polyneuropathy (CIDP) in adults. In 2014 it was also approved for the treatment of primary immunodeficiency (PI) in patients 2 years and older. Hyqvia is infused under the skin (subcutaneously) up to once every 4 weeks.

CIDP is a rare, immune-mediated neuromuscular disorder with symptoms such as weakness, tingling or loss of feeling in the arms and legs, and trouble walking.
Hyqvia contains immune globulin (IG) antibodies collected from human plasma that helps to protect against infection and disease. Hyaluronidase is a genetically designed protein used to help the body absorb other injected medications.
The mechanism of action of immune globulin in the treatment of CIDP in adults is not fully known but may include effects on the immune system. Hyqvia is for subcutaneous use only.
Immune globulin (IG) therapy is the guidelines-based standard of care for maintenance treatment in CIDP, but intravenously IG (IVIG)can be challenging for patients due to infusion duration, drug volumes or venous access. Hyqvia offers a subcutaneous (under the skin) infusion option for patients and caregivers that can be given by a healthcare provider or at home after appropriate training.
Approval was based on the Phase 3 ADVANCE-CIDP 1 study. Patients were randomized to receive either Hyqvia or placebo at the same dose and infusion frequency as their prior IVIG treatment (every 2, 3 or 4 weeks) for 6 months or until withdrawal or relapse. Results showed a statistically significant difference in relapse rate in favor of Hyqvia compared to placebo at 6 months.
The most common side effects reported in studies include headache, vomiting, fatigue, nausea, fever, itching, redness, stomach area (abdominal) pain.
Takeda’s Gammagard Liquid [Immune Globulin Infusion (Human) 10% Solution] intravenous immunoglobulin (IVIG) therapy was also approved at the end of January to improve neuromuscular disability and impairment in adults with CIDP.

Non-Opioid Zynrelef Indications Expanded in Post-Surgical Pain Relief

The FDA has approved Zynrelef (bupivacaine and meloxicam) extended-release solution for soft tissue and orthopedic surgical procedures including foot and ankle, and other procedures in which direct exposure to articular cartilage is avoided. It is indicated in adults for postsurgical analgesia for up to 72 hours.

Zynrelef, from Heron Therapeutics, was previously approved for foot and ankle, small-to-medium open abdominal, and lower extremity total joint arthroplasty surgical procedures in adults.
It is an extended-release, fixed-dose combination of the local anesthetic bupivacaine and the nonsteroidal anti-inflammatory drug (NSAID) meloxicam indicated for the management of postoperative pain. In some patients, it may eliminate or lower the need for opioids after surgery.
Zynrelef is intended for single-dose administration only and given by instillation into the surgical site before closure.
As Zynrelef contains the NSAID meloxicam, the product label carries a boxed warning for an increased risk of cardiovascular and gastrointestinal events.
The most common side effects include vomiting (in soft tissue procedures) and constipation and headache (in orthopedic procedures).

Posted January 2024

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