FDA Action Alert: BMS, Marinus, Merck and More

FDA_Sarah Silbiger/Getty Images

Sarah Silbiger/Getty Images

The U.S. Food and Drug Administration has a full PDUFA calendar for the rest of March for New Drug Applications and Biologics License Applications. Here’s a look at the next two weeks.

Bristol Myers Squibb’s Relatlimab and Nivolumab for Metastatic Melanoma

Bristol Myers Squibb has a target action date of March 19, 2022, for its Biologics License Application for relatlimab and nivolumab (Opdivo) fixed-dose combination for patients 12 years and older with unresectable or metastatic melanoma. The application is built on the Phase II/III RELATIVITY-047 trial data. Relatlimab is the company’s anti-LAG-3 antibody, and Opdivo is a checkpoint inhibitor of PD-1.

The RELATIVITY-047 Phase II/III trial evaluated the fixed-dose combination compared to Opdivo alone in patients with previously untreated metastatic or unresectable melanoma. The trial met its primary endpoint of progression-free survival (PFS).

Marinus’ Ganaxolone for Seizures Associated with CCD, a Rare, Genetic Epilepsy

Marinus Pharmaceuticals has a target action date of March 20 for its New Drug Application (NDA) for ganaxolone for treatment of seizures associated with CDKL5 deficiency disorder (CDD), a rare, genetic epilepsy. It is being reviewed under Priority Review designation. It also received orphan drug designation and Rare Pediatric Disease (RPD) designation. If the NDA is approved, the company is eligible to receive an RPD Priority Review Voucher that can be sold or transferred.

The NDA is based on data from the Phase III Marigold trial. Patients receiving the drug demonstrated a 30.7% mean reduction in 28-day major motor seizure frequency compared to 6.9% in the placebo group.

Merck’s Gefapixant for Chronic Cough

Merck had a target action date of March 21 for its NDA for gefapixant for the treatment of refractory chronic cough or unexplained chronic cough in adults. The drug is an oral, selective P2X3 receptor antagonist. The agency initially accepted the NDA on March 1, 2021, with a PDUFA date of December 21, 2021. On January 24, 2022, the company indicated the FDA had issued a Complete Response Letter (CRL), requesting additional information related to the measurement of efficacy. A week earlier, the drug was approved by the Japan Ministry of Health, Labor and Welfare (MHLW) under the name Lyfnua for both indications.

The NDA was based on results from the COUGH-1 and -2 Phase III trials in patients with RCC or UCC. In COUGH-1 and COUGH-2, the efficacy outcomes measures were 24-hour cough frequency at week 12 and 24-hour cough frequency at week 24, respectively.

TherapeuticsMD’s Bijuva for Menopausal Hot Flashes

TherapeuticsMD has a target action date of March 21 for its supplementary NDA for Bijuva (estradiol and progesterone) capsules for moderate to severe vasomotor symptoms — hot flashes or flushes — due to menopause in women with a uterus. The drug is the only FDA-approved bio-identical hormone therapy combination of estradiol and progesterone in a single, oral capsule. It is taken once daily for moderate to severe vasomotor symptoms due to menopause in women with a uterus. This sBLA is for a new low dose of the drug.

TG Therapeutics’ Ublituximab and Ukoniq for CLL and SLL

TG Therapeutics had a target action date of March 25 for its BLA for its ublituximab in combination with Ukoniq (umbralisib) for chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL). Ublituximab is an investigational glycoengineered anti-CD20 monoclonal antibody. Ukoniq (umbralisib) is a once-daily, oral, inhibitor of PI3K-delta and CK1-epsilon.

The BLA was based on the Phase III UNITY-CLL trial data, where the primary endpoint was superior PFS for the U2 combination compared to the control arm. It met the primary endpoint.

On March 3, TG announced the FDA had extended the PDUFA date to June 25, 2022, for the BLA and sNDA for these indications. The FDA indicated the updated overall survival analyses the company submitted in February 2022 was a major amendment and required for a full review.

Zogenix’ Fintepla for Seizures Associated with Lennox-Gastaut Syndrome

Zogenix has a target action date of March 25 for its supplemental NDA for Fintepla (fenfluramine) for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS). It was under Priority Review. The submission was based on the Phase III Study 1601 in 263 patients. It demonstrated the drug was superior to placebo in reducing the frequency of drop seizures and long-term safety and effectiveness data from the drug’s ongoing open-label extension trials. The FDA approved the drug in June 2020 for seizures associated with Dravet syndrome in patients two years of age and older.

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