Date: 2017-05-22
Type of information: Granting of the orphan status in the US
Product name: PF-06939926 (mini-dystrophin) Recombinant adeno-associated virus serotype 9 vector expressing codon-optimized miniaturized version of DMD gene
Compound: adeno-associated viral vector serotype 9 containing the human mini-dystrophin gene
Therapeutic area: Rare diseases - Genetic diseases - Neuromuscular diseases
Action mechanism:
- gene therapy. PF-06939926 is an investigational, recombinant, adeno-associated virus, serotype 9 (AAV9) carrying a truncated human dystrophin gene (mini-dystrophin) under the control of a human muscle specific promotor. This gene therapy has been developed by Bamboo Therapeutics, an Asklepios BioPharmaceutical's spin-out. Pfizer acquired the company in August 2016. Richard Jude Samulski, PhD, AskBio founder and Chief Scientific Officer,and Executive Chairman of Bamboo, remained with Pfizer through February 2018 to further accelerate the asset’s development.
Company: Pfizer (USA - NY)
Disease: Duchenne muscular dystrophy
Latest news:
- • On May 22, 2017, the FDA has granted orphan drug designation for PF-06939926 (mini-dystrophin/ecombinant adeno-associated virus serotype 9 vector expressing codon-optimized miniaturized version of DMD gene) for treatment of Duchenne muscular dystrophy.
- • On 11-13 July, 2016, the Committee for Orphan Medicinal Products (COMP) has recommended the granting of an orphan designation for adeno-associated viral vector serotype 9 containing the human mini-dystrophin gene for treatment of Duchenne muscular dystrophy.
Patents:
Submission of marketing authorization application USA :
Submission of marketing authorization application UE:
Withdrawal of marketing authorization application USA:
Withdrawal of marketing authorization application UE:
US authorization:
UE authorization:
Favourable opinion UE:
Favourable opinion USA:
Orphan status USA: 2017-05-22
Orphan status UE: 2016-08-29
Pediatric exclusivit _USA:
Pediatric exclusivity UE:
OTC status:
Other news:
Is general: Yes
