Inspiration Biopharmaceuticals, Inc., has submitted a Biologics License Application (BLA) to the US Food and Drug Administration (FDA) for the approval of IB1001, an intravenous recombinant factor IX (rFIX) protein for the treatment and prevention of bleeding in individuals with haemophilia B.

Based on the terms of its agreement with Ipsen, Inspiration will receive a $35 million milestone payment associated with its filing of the BLA. In return, Inspiration will issue a convertible note to Ipsen, bringing Ipsen’s fully diluted equity ownership position in Inspiration to approximately 43.5 per cent.

Approval of IB1001 would represent a significant advance for the haemophilia community, including the estimated 75 per cent of people living with haemophilia worldwide who do not have adequate access to currently available treatments. IB1001 would be the first recombinant therapy for people with haemophilia B introduced in more than 15 years.

Regulatory review is now pending in both the US and Europe. Inspiration’s Marketing Authorization Application for IB1001 was accepted by the European Medicines Agency in September 2011. The company is currently finalizing plans for additional regulatory filings and preparing for the commercial launch of IB1001.

“The introduction of recombinant factor IX was an important step forward in the treatment of haemophilia B, but a single product is not sufficient to meet the needs of all people affected by this disease,” said John P Butler, Inspiration’s chief executive officer. “At Inspiration, we are solely focused on the needs of people with haemophilia, and we are proud of the progress we have made towards bringing forward a new choice of treatment for patients. We plan to work closely with regulatory authorities both here in the US and around the world to make IB1001 available as broadly and rapidly as possible.”

The IB1001 BLA filing includes a comprehensive set of pharmacokinetics safety, and efficacy data from a Phase 3 clinical trial in people affected by haemophilia B. A surgery substudy was also included.

John Taylor, co-founder and chairman of Inspiration, stated, “With sons affected by haemophilia B, Inspiration co-founder Scott Martin and I are acutely aware of the impact of limited treatment options on families. We believe more choice can help us achieve our mission to broaden access to care—increasing the supply of products, driving healthy competition, and supporting prophylaxis, which is rapidly becoming the standard of care in haemophilia. We believe that the approval of IB1001 will be the first step in our journey to make broader access to care a reality for thousands of individuals with haemophilia.”

IB1001 is an intravenous rFIX product being developed for the treatment and prevention of bleeding in individuals with haemophilia B. IB1001 has completed pivotal phase III clinical studies that support the filling of the BLA, conducted at study sites in the US, Europe and India.

To date, IB1001 has been well tolerated by patients and pharmacokinetic (PK) analyses have demonstrated non-inferiority to the one approved rFIX product currently marketed for the treatment of haemophilia B. Confirmatory PK studies reported at the 53rd Annual Meeting of the American Society of Haematology showed no evidence of development of inhibitors during treatment periods ranging from 4 to 18 months.

Results recently presented at the 5th Annual Congress of the European Association for Haemophilia and Allied Disorders demonstrated that IB1001 provided effective surgical haemostasis in study participants with haemophilia B undergoing major surgical procedures.

Inspiration Biopharmaceuticals is exclusively dedicated to developing treatments for haemophilia, with a primary mission to broaden access to care by providing safe and effective recombinant therapies and advancing innovation for people living with haemophilia.