TSHA - FDA to back accelerated approval pathway for gene therapies

2023-03-21 07:43:24 ET

A top FDA official said Monday that the agency needs to consider accelerated approval, a regulatory avenue commonly used to expedite the market entry of cancer drugs, for gene therapies.

Dr. Peter Marks, who heads the FDA's biologics unit, said that the agency will support the use of measurable biomarkers in gene therapy clinical studies as substitutes for other biological indicators to help developers secure "accelerated approval."

The FDA's accelerated approval program focuses on surrogate endpoints such as radiographic images and physical signs that are believed to predict clinical benefit but are not direct measures of a clinical benefit.

The program accelerates the market entry of drugs for unmet medical needs as the use of surrogate endpoints cuts the approval time.

"The FDA views gene therapy as an excellent opportunity to expedite the delivery of potentially life-saving therapies to patients with rare diseases," Dr. Marks, the director of FDA's Center for Biologics Evaluation and Research, said.

His comments come days after Sarepta Therapeutics ( NASDAQ: SRPT ), which seeks accelerated approval for its gene therapy SRP-9001 in Duchenne muscular dystrophy, said that the FDA plans to hold an AdCom meeting to discuss the approvability of the treatment.

Other leading gene therapy developers: bluebird bio ( BLUE ), CRISPR Therapeutics ( CRSP ), Iovance Biotherapeutics ( IOVA ), uniQure ( QURE ), Taysha Gene Therapies ( TSHA ), BioMarin Pharmaceutical ( BMRN ), REGENXBIO ( RGNX )

A potential market entry of SRP-9001 will help accelerate Sarepta's ( SRPT ) "strong" growth trends, Seeking Alpha contributor Biologics argued recently.

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