FDA Action Alert: Blueprint, Orphazyme and Eton


The month of June continues to be busy for the U.S. Food and Drug Administration (FDA). There are three PDUFA dates on the calendar for this week. Here’s a look.

Blueprint Medicines’ Avapritinib for Advanced Systemic Mastocytosis

Blueprint Medicines has a target action date of June 16, 2021, for its supplemental New Drug Application (sNDA) for avapritinib for adults with advanced Systemic Mastocytosis (SM). SM is a rare hematologic disorder that results in an overproduction of mast cells and the accumulation of mast cells in the bone marrow and other organs. This can lead to a range of debilitating symptoms, organ dysfunction and failure. Almost all cases of SM are driven by the KIT D816V mutation, which activates mast cells abnormally. Avapritinib is marketed as Ayvakit for unresectable or metastatic gastrointestinal stromal tumor (GIST) patients with a mutation called PDGFRA exon 18.

The submission was built on data from the registrational PATHFINDER trial in advanced SM. The trial demonstrated an overall confirmed response rate of 75%. It also included data from the PIONEER Part 1 trial that highlighted the impact of Ayvakit on skin manifestations in non-advanced SM.

In the company’s first-quarter report on April 29, Jeff Albers, chief executive officer of Blueprint, said, “With a potential U.S. approval for Ayvakit in advanced systemic mastocytosis in the second quarter, we are looking forward to welcoming a new era of precision medicines care for people living with this debilitating disease.”

Orphazyme’s Arimoclomol for Nieman-Pick Disease Type C

Orphazyme A/S has a target action date of June 17 for its NDA for arimoclomol to treat Niemann-Pick Disease Type C. Originally there was a PDUFA date in March, but the FDA extended the review period under a standard extension to complete the NDA review. The NDA remains under Priority Review and does not impede eligibility for a Pediatric Rare Disease Priority Review Voucher. The drug had previously received the FDA’s Fast-Track and Breakthrough Therapy Designations for NPC as well as Orphan Drug and Rare Pediatric Disease Designations.

Niemann-Pick disease Type C is a rare, genetic, progressively debilitating, and often fatal neurovisceral disease. It is part of a family of lysosomal storage diseases and is caused by mutations that result in a defective NPC protein. This results in lipids normally cleared by the lysosome to accumulate in various organs, including the brain.

Arimoclomal amplifies the production of heat-shock proteins (HSPs). These proteins can rescue defective misfolded proteins, clear protein aggregates, and improve lysosome function. It is an oral drug that can cross the blood-brain barrier. It is also being developed for Gaucher disease, sIBM, and ALS.

Eton’s Ephedrine Ready-to-Use Injection

Eton Pharmaceuticals has a target action date of June 18 for its NDA for ephedrine Ready-to-Use (RTU) injection. Eton has publicly said very little about this NDA, which is the third of four NDAs with PDUFA dates from the company and/or its partners from May 27 through August 6. RTU ephedrine injection is typically used to treatment hypotension during anesthesia. The company generally focuses on developing and commercializing treatments for rare diseases. The other PDUFA dates are May 27 (dehydrated alcohol injection); May 29 (zonisamide oral suspension); and August 6 (topiramate oral solution). On May 28, the company received a Complete Response Letter from the FDA over its NDA for dehydrated alcohol injection for the treatment of methanol poisoning. The CRL reported that the agency had completed the review but determined it could not be approved in its current form.

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