Orphan status for haemophilia gene therapy
pharmafile | January 4, 2012 | News story | Research and Development | AMT, gene therapy, haemophilia
Amsterdam Molecular Therapeutics has been granted an orphan drug designation for its gene therapy for haemophilia B.
The FDA’s orphan designation for the US could provide the company with up to seven years market exclusivity, if the drug is approved, and follows on from European orphan designation in November.
The news is encouraging for the Netherlands based company, which suffered a major setback in October. Its shares tumbled when the European Medicines Agency rejected its lead product Glybera, a gene therapy for a rare disorder called lipoprotein lipase deficiency (LPLD).
AMT is among the very first companies to file gene therapies, but the cutting edge nature of the field makes it a high-risk venture.
The company’s haemophilia B programme uses the same adeno-associated viral (AAV) vector technology as Glybera to replace the missing gene in haemophilia B. In the case of haemophilia B, the missing gene is human factor IX gene, and the treatment is being investigated in a Phase I/II study conducted by St. Jude’s Children’s Research Hospital in Memphis, USA and University College London in the UK.
Promising data from an initial six patients, recently published in the New England Journal of Medicine shows that gene therapy reduced the need for protein replacement treatment, the standard care for haemophilia patients. AMT is preparing for further clinical development work to establish safety, tolerability and proof-of-concept with a factor IX gene therapy produced using its proprietary AAV production system.
“US orphan designation provides additional support for our haemophilia B gene therapy programme and supplements the designation in the EU received in November,” said Jorn Aldag, chief executive of AMT. “The early clinical success seen with the programme to date by our partners is very encouraging. We will build on this success in the coming months.”
Andrew McConaghie
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