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DNA helix
The US Food and Drug Administration (FDA) has accepted an investigational new drug application for SHP654 (also known as BAX 888) and granted the therapy orphan drug designation.
SHP654 is an investigational factor VIII (FVIII) gene therapy intended to treat hemophilia A using an adeno-associated virus serotype 8 vector to deliver codon-optimized, B-domain-deleted FVIII specifically to a patient’s liver, where FVIII would then be produced and used to manage bleeds.
Shire, the company developing SHP654, received FDA clearance for an investigational new drug application to initiate a global, phase 1/2 study of SHP654.
In this study, researchers will evaluate the safety and optimal dose of SHP654 needed to boost FVIII activity levels and affect hemophilic bleeding. Shire expects this study will begin by the end of this year.
The FDA also granted orphan designation to SHP654. The agency grants orphan designation to products intended to treat, diagnose, or prevent diseases/disorders that affect fewer than 200,000 people in the US.
The designation provides incentives for sponsors to develop products for rare diseases. This may include tax credits toward the cost of clinical trials, prescription drug user fee waivers, and 7 years of market exclusivity if the product is approved.
“This important orphan drug designation highlights Shire’s commitment to patients with rare diseases, and, for hemophilia patients specifically, our aim is to help them achieve zero bleeds,” said Paul Monahan, MD, senior medical director of gene therapy at Shire.
“We know that hemophilia care is not one-size-fits-all and that every patient is unique, which is why we continue to focus on optimizing personal outcomes for hemophilia patients by developing innovations to transform care.”
