Products

Date: 2017-03-01

Type of information: Granting of a Fast Track status

Product name: SPK-9001 - adeno-associated viral vector composed of a bioengineered AAV capsid and a codon-optimized expression cassette encoding a high-specific activity variant of human coagulation factor IX

Compound: adeno-associated viral vector composed of a bioengineered AAV capsid and a codon-optimized expression cassette encoding a high-specific activity variant of human coagulation factor IX

Therapeutic area: Rare diseases - Genetic diseases - Hematological diseases

Action mechanism: gene therapy. SPK-9001  is an adeno-associated viral vector composed of a bioengineered AAV capsid and a codon-optimized expression cassette encoding a high-specific activity variant of human coagulation factor IX.

Company: Spark Therapeutics (USA - PA) Pfizer (USA - NY)

Disease: hemophilia B

Latest news:

• • On March 1, 2017,  Spark Therapeutics and Pfizer announced that SPK-9001 has been granted support through the European Medicines Agency (EMA) PRIority MEdicines (PRIME) program. According to the EMA, PRIME is intended to enhance support for new treatments in development "that may offer a major therapeutic advantage over existing treatments, or benefit patients without treatment options."
• SPK-9001 is currently being investigated in an ongoing Phase 1/2 trial for the treatment of hemophilia B.
• •  On July 21, 2016, Spark Therapeutics  and Pfizer announced that the FDA has granted breakthrough therapy designation to SPK-9001 in development for the treatment of hemophilia B. SPK-9001 is being investigated in an ongoing phase 1-2 trial as a potential one-time therapy.
• • On September 9, 2015, the FDA has granted orphan drug designation for adeno-associated viral vector composed of a bioengineered AAV capsid and a codon-optimized expression cassette encoding a high-specific activity variant of human coagulation factor IX for the treatment of hemophilia B.

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Is general: Yes