Selecta Biosciences Inc. (NASDAQ:SELB) and Asklepios Biopharmaceuticals Inc. (AskBio) have announced receipt of Orphan Drug designation for MMA-101 from the US FDA. MMA-101 is an AAV-based gene therapy the company is developing for isolated methylmalonic acidemia due to methylmalonyly-Coa mutase gene mutations treatment. In October 2020, the FDA granted MMA-101 rare pediatric disease designation.
MMA-101 combination with ImmTOR for treatment of MMA patients
Sheila Mikhail, AskBio’s CEO and Co-founder said that the orphan drug designation and rare pediatric disease designation for MMA-101 support the huge medical need existing for MMA patients. She said that they are looking forward to collaborating with the FDA to deliver innovative treatment to patients as soon as possible.
Selecta Biosciences’ CEO Carsten Brunn said that ImmTOR’s combination with AAV-gene therapy seeks to address a huge unmet medical need for MMA patients. Brunn said that they are delighted that the agency has acknowledged Selecta and AskBio’s efforts by granting the program Orphan Drug designation. He added that they are pleased to continue the ImmTOR platform’s evaluation to induce AAV-specific tolerance as well as enable gene therapy redosing.
Selecta and AskBio to commence MMA-101 study
MMA is a monogenic disorder whereby the body is unable to breakdown specific fats and proteins. The rare metabolic disease can result in metabolic crisis and often it is linked with long-term complications including intellectual impairment, developmental delays, optic nerve atrophy, chronic kidney diseases, and osteopenia. MMA symptoms are visible in early infancy and will vary from mild to life-threatening and without treatment, the disorder can result in coma or death in some cases. Selecta and AskBio plan to commence phase 1 clinical study of MMA-101 and ImmTor for MMA patients in 1H 2021.
The Orphan Drug designation is for investigational treatments addressing rare diseases that affect less than 200,000 people in the US. The program seeks to encourage the development of rare diseases’ treatment and includes benefits such as application fee waivers and tax credit. On the other hand, the rare pediatric disease designation seeks to incentivize the development of novel treatments for life-threatening diseases.