FDA updates for Week of March 6, 2023


The FDA has approved a novel nasal spray for migraine and a generic leukemia treatment. The agency has granted priority review to Leqembi for traditional approval but extended the review of hemophilia A gene therapy. Regulators have also assigned target dates for Voxzogo in young children with dwarfism, a skin infection gel, a nerve disorder drug and for Jardiance in children 10 to 17 with diabetes.

FDA approves novel nasal spray for migraine.

The FDA has approved Pfizer’s Zavzpret (zavegepant) to treat adult patients with migraine with or without aura. Nearly 40 million people in the United States suffer from migraine. Zavzpret is the first calcitonin gene-related peptide (CGRP) receptor antagonist nasal spray. It was developed by Biohaven, which Pfizer acquired in October 2022 for $11.6 billion in cash.

Pfizer indicated that pricing of Zavzpret will be available at launch, which is anticipated in July 2023, and is expected to be comparable in price to other FDA approved CGRP migraine medicines. The FDA approval is based on two pivotal randomized, double-blind, placebo-controlled studies. In these studies, Zavzpret was statistically superior to placebo on the co-primary endpoints of pain freedom and freedom from most bothersome symptom at two hours post-dose. The pivotal study also demonstrated pain relief as early as 15 minutes in a prespecified secondary endpoint versus placebo.

FDA greenlights generic leukemia treatment.

The FDA has approved Shorla Oncology’s generic nelarabine injection to treat adult and pediatric patients one year and older with T-cell acute lymphoblastic leukemia and T-cell lymphoblastic lymphoma. T-cell leukemia is an aggressive blood and bone marrow cancer that progresses quickly. While most leukemias target older people, T-cell leukemia is most common among children. The product is a generic of Novartis’s Arranon.

A spokesperson for Shorla said the company could not comment on wholesale acquisition cost at this time. In preparation for the immediate commercial launch of Nelarabine Injection, Shorla is working with its commercialization services partner, Eversana, which it announced August 2021.

FDA extends review of hemophilia A gene therapy.

The FDA has extended the review of BioMarin Pharmaceutical’s biologics license application (BLA) for Roctavian (valoctocogene roxaparvovec) gene therapy for adults with severe hemophilia A. The new PDUFA target action date is June 30, 2023. Hemophilia A is a genetic disease caused by the deficiency of clotting factor VIII. It is the most common type of hemophilia and occurs much more frequently in males; incidence is estimated at 1 in 4,000-5,000 male births.

BioMarin’s gene therapy uses an adeno-associated virus vector to deliver a functional gene to the cells in the liver that is designed to enable the body to produce Factor VIII on its own without the need for continued hemophilia prophylaxis.

The FDA determined that the company’s submission of the three-year data analysis from the ongoing phase 3 GENEr8-1 study is a major amendment due because of the amount of additional data. The company had anticipated that extra time would be needed for review when it resubmitted its BLA in October 2022 following a complete response letter issued on Aug. 18, 2020

FDA grants priority review to Leqembi for traditional approval.

The FDA has accepted Eisai’s supplemental biologics license application (sBLA) for Biogen/Eisai’s Leqembi (lecanemab-irmb) to convert the accelerated approval to a traditional approval to treat patients with Alzheimer’s disease. The FDA has assigned a Prescription Drug User Fee Act (PDUFA) action date of July 6, 2023. The FDA is currently planning to hold an advisory committee but has not yet announced the date of the meeting.

Last week, the Institute for Clinical and Economic Review (ICER), in an updated evidence report, calculated a health-benefit price benchmark (HBPB) of between $8,900 and $21,500 per year, which is lower than the Leqembi’s wholesale acquisition cost of $26,500 a year. ICER analysts said that Leqembi would need a 66% to 19% discount from its wholesale acquisition cost to fall within commonly used cost-effectiveness thresholds.

The FDA issued an accelerated approval for Leqembi in January 2023. Leqembi it is a humanized immunoglobulin gamma 1 (IgG1) monoclonal antibody approved to treat patients with mild cognitive impairment or mild dementia. The accelerated approval was based on phase 2 data that demonstrated that Leqembi reduced the accumulation of amyloid plaque in the brain. The FDA has determined that the results of phase 3 Clarity AD can serve as the confirmatory study.

FDA assigns target date for Voxzogo in young children with dwarfism.

The FDA has accepted the BioMarin Pharmaceutical’s supplemental new drug application (sNDA) for Voxzogo (vosoritide) for injection to expand treatment in the United States to include children with achondroplasia under the age of 5. Achondroplasia is the most common form of dwarfism. The FDA has set a PDUFA target action date of Oct. 21, 2023, for the sNDA.

In patients with achondroplasia, endochondral bone growth, an essential process by which bone tissue is created, is affected by a mutation in fibroblast growth factor receptor 3 gene (FGFR3). The worldwide incidence rate of achondroplasia is about one in 25,000 live births.

Voxzogo, a C-type natriuretic peptide (CNP) analog, acts as a positive regulator of the signaling pathway downstream of FGFR3 to promote endochondral bone growth. It received accelerated approved by the FDA to increase growth in pediatric patients with achondroplasia who are 5 years of age and older with open epiphyses, which are growth plates that haven’t fully formed. As of December 2021, Voxzogo’s list price was $8,990 for 10 single-dose vials. BioMarin offers a copay assistance program that covers up to $17,000 a year.

The sNDA is supported by results from a phase 2 clinical trial, which demonstrated similar safety and efficacy profiles in children under 5 years of age as compared with those ages 5 years and older.

FDA sets goal date for skin infection gel.

The FDA has accepted Novan’s new drug application (NDA) seeking approval for berdazimer gel (SB206) to treat the skin infection molluscum contagiosum, an infection caused by a poxvirus. It is usually benign with mild skin disease characterized by lesions that may appear anywhere on the body. Within 6 to 12 months, Molluscum contagiosum typically resolves without scarring but may take as long as four years, according to the CDC. It affects about 6 million people — mostly children — in the United States. Molluscum is transmitted by human contact or via a contaminated object, such as a towel at the pool or an athletic mat.

Berdazimer sodium is a new chemical entity that releases nitric oxide and has anti-viral activity. The Prescription Drug User Fee Act (PDUFA) goal date is Jan. 5, 2024. If it is approved, the pharma maker would target launching in the first half of 2024.

FDA sets action date for nerve disorder drug.

The FDA has accepted for review a new drug application (NDA) for eplontersen, an investigational antisense medicine to treat patients living with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN). The application has been given a Prescription Drug User Fee Act (PDUFA) action date of Dec. 22, 2023.

Patients with ATTRv-PN experience ongoing debilitating nerve damage throughout their body resulting in the progressive loss of motor function. These patients accumulate TTR in other major organs, which progressively compromises their function and eventually leads to death within five to 15 years of disease onset.

Developed by Ionis Pharmaceuticals, eplontersen is an investigational medicine designed to reduce the production of transthyretin (TTR) protein to treat both hereditary and non-hereditary forms of ATTR amyloidosis (ATTR). The FDA has granted eplontersen orphan drug designation. In December 2021, Ionis and AstraZeneca entered into a strategic collaboration to develop and commercialize eplontersen.

FDA accepts sNDA for Jardiance in children 10 to 17 with diabetes.

The FDA has accepted a supplemental new drug application (sNDA) for Jardiance (empagliflozin) to lower blood sugar in children 10 years and older with type 2 diabetes. An FDA decision is expected in the second quarter of 2023. Developed by Boehringer Ingelheim and Eli Lilly, Jardiance is approved for several indications, including treating adults with type 2 diabetes, to reduce the risk of cardiovascular disease in adults with diabetes and to reduce the risk of heart failure. The current list price for a month supply of Jardiance is $570.48, but patients can access a copay card for $10 per month.

The sNDA is based on the results from the DINAMO phase 3 trial, which enrolled 158 patients between 10 and 17 years of age.

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