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FDA Updates for the Week of Feb. 6, 2023


The FDA has approved two pediatric indications this week: Takhyzro for hereditary angioedema and Eylea for retinopathy of prematurity. The agency expanded the label of Cibinqo for atopic dermatitis in adolescents, and granted full approval to Jemperli for endometrial cancer. Additionally, the agency has set PDUFA dates for two drugs: reproxalap for dry eye and zuranolone for depression.

FDA okays pediatric indication for Takeda’s Takhyzro

The FDA has approved the supplemental biologics license application (sBLA) for the expanded use of Takeda’s Takhyzro (lanadelumab-flyo) to prevent attacks of hereditary angioedema (HAE) in pediatric patients 2 to under 12 years old. HAE attacks can involve serious and debilitating swelling in the abdomen, face, feet, genitals, hands and throat, and potentially fatal upper airway angioedema has been reported in patients as young as 3 years of age.

Takhyzro is a fully human monoclonal antibody that specifically binds and decreases plasma kallikrein activity, which plays a critical role in HAE. It was originally approved in the United States in 2018 to prevent attacks of HAE in adult and pediatric patients 12 years and older.

Takhyzro will continue to be priced at parity to its current pricing across the United States. The approximate cost of the 150 mg/1 mL pediatric dose of Takhyzro will be half that of the 2mL adult dose — which is $24,840, according to the company’s website.

The approval was supported by extrapolation of efficacy data from the HELP Study, a phase 3 trial that included patients 12 to under 18 years of age, and additional pharmacokinetic analyses showing similar drug exposures between adults and pediatric patients. It was also based on safety and pharmacodynamic data from the SPRING Study, an open-label phase 3 trial in 21 HAE patients 2 to under 12 years of age. Takhyzro reduced the rate of HAE attacks in pediatric patients by a mean of 94.8% compared with baseline, from 1.84 attacks per month to 0.08 attacks during the 52-week treatment period, according to Takeda. The majority of patients (76.2%) were attack-free with an average of 99.5% attack-free days.

FDA approves first pediatric indication for blockbuster Eylea.

The FDA approved the first pediatric indication for Regeneron’s Eylea (aflibercept) to treat preterm infants with retinopathy of prematurity (ROP). Retinopathy of prematurity is a leading cause of childhood blindness worldwide.

Bayer and Regeneron are collaborating on the global development of Eylea, and Regeneron maintains exclusive rights in the United States.

Eylea is currently available to treat patients with age-related macular degeneration, diabetic macular edema and diabetic retinopathy. It is a VEGF inhibitor that is injected into the eye. It is designed to block the growth of new blood vessels and decrease the ability of fluid to pass through blood vessels in the eye.

The blockbuster Eylea, which realized sales of $6.26 billion in 2022, is now indicated to treat five retinal conditions caused by ocular angiogenesis, Regeneron said. While Regeneron has not released the wholesale acquisition cost (WAC) for the infant dose, the current WAC per prefilled syringe or single-dose vial 2mg/0.05mL is $1,850.

The FDA approval is supported by data from two randomized global phase 3 trials — FIREFLEYE and BUTTERFLEYE — investigating EYLEA 0.4 mg versus laser photocoagulation (laser) in infants with ROP. In both trials, approximately 80% of EYLEA-treated infants achieved an absence of both active ROP and unfavorable structural outcomes at 52 weeks of age, which is better than would have been expected without treatment, Regeneron said. No new safety signals were observed in either trial.

FDA grants full approval to Jemperli for endometrial cancer.

The FDA has converted the accelerated approval of GSK’s Jemperli (dostarlimab-gxly) to a full approval to treat patients with advanced endometrial cancer. It is indicated for patients who have a mismatch repair-deficient (dMMR) cancer that has progressed following a prior treatment. In April 2021, Jemperli received accelerated approval for this indication.

The price of Jemperli is about $11,540 per 500 mg/10 mL vial, which is initially given once every three weeks for four doses, according to Drugs.com. GSK offers co-pay assistance of up of $26,000 for patients with commercial insurance.

The full approval is based on additional data collected from the A1 expansion cohort of the ongoing GARNET trial, a phase I single-arm study of Jemperli monotherapy in patients with advanced or recurrent solid tumors. Cohort A1 evaluated the efficacy of Jemperli in 141 patients with dMMR advanced or recurrent endometrial cancer. Confirmed overall response rate (ORR) was 45.4%, with a 15.6% complete response rate and a 29.8% partial response rate. The most common adverse reactions were fatigue/asthenia, anemia, rash, nausea, diarrhea, constipation, and vomiting. The most common grade 3 or 4 adverse reactions were anemia, increased transaminases, urinary tract infection, fatigue/asthenia, and diarrhea.

FDA expands Cibinqo label for atopic dermatitis in adolescents.

The FDA has approved an additional indication for Pfizer’s Cibinqo (abrocitinib) to include adolescents (12 to <18 years) with refractory, moderate-to-severe atopic dermatitis (AD). It was was previously approved only for the treatment of adults 18 years and older.

Cibinqo is an oral, once-daily, Janus kinase 1 (JAK1) inhibitor. Inhibition of JAK1 is thought to modulate multiple cytokines involved in atopic dermatitis. It has an average retail price of $6,497.80 for 30 tablets, according to GoodRx. Pfizer offers a savings card with as little as $0 out of pocket up to a maximum of $15,000 annually.

The prescribing information was updated to include data from JADE TEEN, a phase 3 trial that supported the expanded indication. It evaluated both the 100 mg and 200 mg doses of Cibinqo versus placebo in adolescents 12 to <18 years of age. In the JADE TEEN trial, the Investigator Global Assessment at week 12 was 39% with Cibinqo 100 mg, 46% with 200 mg, and 24% with placebo. Eczema Area and Severity Index was 64% with the 100 mg dose, 71% with the 200 mg dose and 41% with placebo. Across trials, the most common adverse events reported included nasopharyngitis, nausea, and headache.

FDA accepts NDA for zuranolone for depression.

The FDA has accepted the new drug application (NDA) for zuranolone in to treat patients with major depressive disorder and postpartum depression. The application has been granted priority review and the FDA has assigned a Prescription Drug User Fee Act (PDUFA) action date of Aug. 5, 2023.

Developed by Sage Therapeutics and Biogen, zuranolone is a rapid-acting neuroactive steroid. In people with depression, it is thought to work by rapidly rebalancing dysregulated neuronal networks to help reset brain function. Zuranolone targets brain networks responsible for functions such as mood, arousal, behavior, and cognition.

The zuranolone NDA includes data from the LANDSCAPE and NEST clinical development programs. The LANDSCAPE program includes five studies of zuranolone in adults with major depressive disorder (MDD-201B, MOUNTAIN, SHORELINE, WATERFALL, and CORAL)

FDA sets PDUFA date for dry eye disease drug.

The FDA has accepted the new drug application (NDA) for topical ocular reproxalap, a first-in-class drug to treat patients with the signs and symptoms of dry eye disease. The FDA assigned a Prescription Drug User Fee Act (PDUFA) date of Nov. 23, 2023.

Developed by Aldeyra Therapeutics, reproxalap is a first-in-class small-molecule modulator of RASP (reactive aldehyde species), which are elevated in ocular and systemic inflammatory disease. In patients with dry eye disease, RASP may contribute to ocular inflammation and changes in tear lipid composition.

Results of the phase 3 TRANQUILITY-2 trial, which were released in June 2022, showed that reproxalap was statistically superior to vehicle for each of the two prespecified primary endpoints, Schirmer test and ≥10 mm Schirmer test responder proportions after a single day of dosing. The Schirmer test, a measure of ocular tear production, is the objective sign most commonly used for drug approval for dry eye disease.

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