Spark and Pfizer's haemophilia gene therapy gets European PRIME status

Spark and Pfizer's haemophilia B gene therapy SPK-9001 has been granted access to the Priority Medicines (PRIME) programme by the EMA.  

The treatment is in the vanguard of a new generation of gene therapies, and could effectively provide a cure for some patients with the life-threatening bleeding condition.

However the drug is still in an early stage phase 1/2 trial, and the PRIME status is aimed at helping create a pathway to market for the treatment, one of the first of its kind.

The FDA granted the drug a Breakthrough Therapy designation seven months ago, and the EMA's PRIME programme the EMA's equivalent progamme aimed at accelerating and assist development of highly promising innovative treatments.

SPK-9001 is an adeno-associated virus (AAV) therapy that introduces the gene responsible for producing factor IX protein to the liver.

In its most recent data, a single infusion with SPK-9001 resulted in one patient reducing their number of factor IX infusions to zero without experiencing any bleeds, one year after infusion.

A further seven of the nine participants experiencing consistent factor IX activity, 12 weeks post-SPK-9001 infusion; and, overall, no participants developing factor IX inhibitors or experiencing thrombotic events after SPK-9001 administration.

No serious adverse events have so far been reported, however two trial participants have experienced an immune response to the virus. These cases spooked investors, despite Spark saying that corticosteroid treatment brought these patients back under control.

The support from the PRIME Programme could help to reduce the development and evaluation time for SPK-9001 and allow earlier patient access to the drug.

Factor IX is the key molecule responsible for blood clotting and is missing in people with haemophilia B. The only current therapeutic option for people with haemophilia B is to have regular infusions of factor IX.

[caption id="attachment_25502" align="alignnone" width="180"] Spark's Jeffrey Marrazzo[/caption]

Jeffrey Marrazzo, chief executive officer of Spark Therapeutics, welcomed the PRIME status and said it could help the treatment reach patients faster.

“We appreciate the EMA’s dedication to moving treatments forward for patients who are underserved with current options, and we look forward to our continued collaboration on SPK-9001.”

One of the company's other investigational candidates, SPK-RPE65 – itself the recipient of Breakthrough Therapy designation in 2014 - is showing promise restoring vision in people with inherited retinal disease (IRD).

Initial clinical data for its haemophilia A candidate, SPK-8011, is expected to be released in the first half of this year.