Beren Therapeutics is seeking approval in Neimann-Pick disease type C, an application the FDA was set to decide on by August 17. The new target action date is November 17.
The FDA has pushed back its decision date for Beren Therapeutics’ intrathecal injection for infantile-onset Niemann-Pick disease type C.
The regulator accepted Beren’s data package for priority review in February, giving an initial target action date of August 17. Then, in March, Beren filed additional data and documentation in response to a previous FDA request.
The agency considered this submission to be a major adjustment to the original drug application, necessitating an extension, according to the company’s release on Thursday. The new decision data is November 17.
Niemann-Pick disease type C (NPC) is a rare and severe neurodegenerative disorder that arises from pathogenic mutations to the NPC1 or NPC2 genes, impairing the movement of cholesterol molecules inside cells. Patients with NPC suffer from progressive neurological decline and early death.
Beren’s answer to NPC is adrabetadex, a proprietary mixture of various isomers of the oligomer 2-hydroxypropyl-β-cyclodextrin. The drug candidate, according to the company, can restore cholesterol trafficking in the cells.
Beren is specifically proposing adrabetadex for infantile-onset NPC, which refers to cases that manifest in children younger than 6 years of age. This NPC subtype is typically linked to more rapid disease progression and worse prognosis, according to the biotech.
The drug elicited a 71% reduction in the risk of death versus external controls, Beren said in its February news release, noting that this effect was highly statistically significant. The FDA in December last year granted adrabetadex its Breakthrough Therapy designation.
If approved, adrabetadex will share the NPC market with Zevra Therapeutics’ Miplyffa, which became the first-ever drug cleared for the indication in September 2024, and IntraBio’s Aqneursa, which followed just days later. Azafaros is also advancing an NPC therapy dubbed nizubaglustat. The molecule entered late-stage development last July.
Beren’s delay comes just days after the FDA similarly extended its review of AstraZeneca’s breast cancer pill camizestrant, citing the need for more time to review additional submissions. The pharma is developing camizestrant, in combination with the AKT blocker Truqap, as a first-line therapy for patients with HER2-negative, HR-positive, ESR1-mutated breast cancer.
It is unclear whether the exits of FDA Commissioner Marty Makary and several other senior leaders in recent weeks contributed to the delays.
