Regulatory Affairs

Shire submits IND for hemophilia A treatment in US

PBR Staff Writer Published 07 July 2017

Shire has filed an investigational new drug (IND) application to the US Food and Drug Administration (FDA) for its gene therapy drug, SHP654, to treat hemophilia A.

SHP654 is an investigational factor VIII (FVIII) gene therapy, which will protect hemophilia A patients against bleeds through delivering long-term and constant level of factor expression.

Shire had acquired technology from Chatham Therapeutics for use in the development of SHP654 investigational gene therapy.

It is a gene therapy intended to treat hemophilia A through utilizing recombinant adeno-associated virus serotype 8 (rAAV8) vector to deliver a codon-optimized and B-domain deleted FVIII (BDD-FVIII) specifically to a patient’s liver.

Shire has submitted the application based on data generated from pre-clinical and phase 1 studies, which demonstrated the potential utility of the investigational treatment.

If FDA accepts the firm’s IND, the company will evaluate SHP654 in a global multi-center trial, which will include the assessment of the treatment to enhance factor VIII activity levels and affect hemophilic bleeding.

According to Shire, Hemophilia A is a rare bleeding disorder that causes longer-than-normal bleeding due to lack of clotting factor VIII in the blood.

Shire gene therapy senior medical director Dr Paul Monahan said: “SHP654 uses a proprietary technology platform designed to produce sustained levels of factor similar to the natural mechanisms of the body.

 “Our goal with gene therapy for hemophilia is to uphold the highest standards for safety and efficacy.”

In June, Shire secured FDA approval for its Mydayis (mixed salts of a single-entity amphetamine product) to control attention deficit hyperactivity disorder (ADHD) in patients 13 years and older.

Mydayis is a once-daily treatment and prescription medicine, which includes three different types of drug-releasing beads to control ADHD in patients 13 years and older.

Image: Shire location in Lexington Massachusetts. Photo: courtesy of John Phelan.