FDA Places ASC618, Gene Therapy for Hemophilia A, on Fast Track

Margarida Maia, PhD avatar

by Margarida Maia, PhD |

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The U.S. Food and Drug Administration (FDA) has given fast track designation to ASC618, an investigational one-time gene therapy for hemophilia A in the pipeline of ASC Therapeutics.

Fast track status works to speed the development and regulatory review of treatments for serious disorders that show a potential to fill an unmet need.

The Committee for Orphan Medicinal Products (COMP), a branch of the European Medicines Agency (EMA), also issued a positive opinion to designate ASC618 an orphan medical product for the EU. This status supports the development and review of potential therapies for serious disorders affecting fewer than 5 in 10,000 people in the EU, and offers 10 years of marketing exclusivity across the EU upon approval.

“We are very pleased with the FDA’s and EMA’s regulatory decisions regarding ASC618. This brings us one step closer for providing a truly novel therapeutic approach for hemophilia A, providing potentially a functional cure for patients who currently require life-long care,” Ruhong Jiang, PhD, CEO of ASC Therapeutics, said in a press release.

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Hemophilia A is caused by the lack of the blood clotting factor VIII (FVIII), a protein whose instructions are provided by F8, a gene that is mutated in people with the disease.

ASC618 is designed to deliver a shortened, but optimized version of the gene to liver cells, which is expected to work better than the full version. It is carried in a harmless vector, called adeno-associated virus variant 8 (AAV8), that the company derived from a naturally occurring virus.

In preclinical studies, ASC618 led to an increase in the amount of FVIII that was at least 10 times that made from the full version of the F8 gene. According to the company, ASC618 may be able to keep a lasting supply of FVIII with relatively low therapeutic doses, while minimizing stress in liver cells that could lower the production of the FVIII protein.

“ASC618 has the potential to reduce therapeutic dosing and increase durability of hemophilia A gene therapy with a novel bioengineered construct that can improve biosynthesis, protein folding, and secretion of factor VIII,” said Oscar Segurado, MD, PhD, chief medical officer at ASC Therapeutics.

The company announced plans to begin clinical testing of ASC618 in hemophilia A patients last year, after the FDA gave the green light for such trials.

“These regulatory milestones culminate years of hard work, dedication, and collaborative efforts by our functional teams,” Segurado added.

The Phase 1/2 trial (NCT04676048) is expected to soon open, and will test the safety, tolerability, and preliminary efficacy of ASC618 — given as a single infusion into the bloodstream — in up to 12 adults with moderate to severe hemophilia A.

The FDA previously designated ASC618 an orphan drug, which provides regulatory support and financial benefits to accelerate the therapy’s clinical development and review, including a seven-year period of marketing exclusivity in the U.S. should it be approved.