FDA Action Alert: Aquestive, BMS, GBT and Strongbridge/Xeris


Although the U.S. Food and Drug Administration (FDA) largely cleared its calendar ahead of the holidays, it still has a couple open PDUFA dates leading up to January 1, 2022. Here’s a look.

Aquestive’s Libervant for Seizure Clusters

Aquestive Therapeutics has a target action date of December 23, 2021, for its New Drug Application (NDA) for Libervant (diazepam) Buccal Film for the management of seizure clusters. It is a resubmission. The company received a Complete Response Letter from the FDA in September 2020, conducted a Type A meeting with the agency in November 2020, and after further guidance in February 2021, added additional statistical modeling and supporting analyses.

Libervant is a soluble film formulation of diazepam, a benzodiazepine intended for rapid treatment of acute uncontrolled seizures in selected, refractory patients with epilepsy on stable regimens of anti-epileptic drugs (AEDs) that require occasional use of diazepam to control increased seizure activity. It is dosed on the inside of the cheek (buccally). The current standard of care rescue therapy is a rectal gel, which is invasive, inconvenient and difficult to administer.

At the company’s third-quarter report on November 2, Keith Kendall, Aquestive’s chief executive officer, said, “We continue to interact with the FDA regarding the NDA submission of Libervant, including responding to several information requests to date, having a recent inspection of our post-marketing adverse event capabilities, regarding changes in language relating to packaging, approval of the product trade name and an update to the patent information included in the resubmission. We are continuing to prepare for commercialization with payer and sales force planning underway.”

BMS’s Orencia for Acute Graft Versus Host Disease

Bristol Myers Squibb had a target action date of December 23 for its Biologics License Application (sBLA) for Orencia (abatacept) for prevention of moderate to severe acute graft versus host disease (aGvHD) in patients six years of age and older receiving unrelated donor hematopoietic stem cell transplantation (HSCT). The agency approved it on December 15. It is the first drug approved by the FDA for this indication. It was approved under Priority Review. The drug is an immunomodulator that disrupts the continuous cycle of T-cell activation.

“By potentially preventing the disease, more patients may successfully undergo bone marrow or stem cell transplantation with fewer complications,” stated Richard Pazdur, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the agency’s Center for Drug Evaluation and Research.

Global Blood Therapeutics’ Oxbryta for Sickle Cell Disease

Global Blood Therapeutics (GBT) had a target action date of December 25 for its Oxbryta (voxelotor) for sickle cell disease (SCD) in children ages four to 11 years of age. On December 17, the FDA approved the drug for that indication under an accelerated approval pathway. It expanded the previously approved use to treat SCD in children 12 years and older. The agency also approved GBT’s separate NDA for the Oxbryta tablets for oral suspension, a new dispersible, once-daily tablet dosage suitable for children ages four to less than 12 as well as older patients who have problems swallowing whole tablets.

“For decades, the sickle cell disease community has been profoundly underserved, and there have been limited treatment options for younger patients with their whole lives ahead of them,” said Ted W. Love, president and chief executive officer of GBT. “Complications of SCD that can cause irreversible organ damage are known to begin in the first few years of life, which is why earlier intervention is critical.”

Strongbridge’s Recorlev for Endogenous Cushing’s Syndrome

Strongbridge Biopharma has a target action date of January 1, 2022, for its NDA for Recorlev (levoketoconazole) for endogenous Cushing’s syndrome. The submission was based on the data from the Phase III SONICS and LOGICS studies. Cushing’s syndrome is a rare, serious and potentially lethal endocrine disease caused by chronic elevated cortisol exposure, often from a benign tumor of the pituitary gland.

The company was acquired by Xeris Biopharma in October. As of Xeris’ third-quarter report on November 10, they are “intensely preparing for the potential commercial launch of Recorlev.”

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