Mustang Bio Inc. (NASDAQ:MBIO) has been awarded Orphan Drug Designation for MB-207, its lentiviral gene therapy for treating patients with X-linked severe combined immunodeficiency which is also called bubble boy disease. The gene therapy is for patients that have been treated previously with hematopoietic stem cell transplantation with indicated retreatment.
Mustang to benefit from Orphan Drug Designation Incentives
Mb-207 received Rare Paediatric Disease Designation on August 2020 to treat patients with XSCID that had previously been treated with HSCT. Usually, the FDA will grant Orphan Drug Designation to therapies meant for effective and safe treatment, prevention, or diagnosis of rare disorders or diseases affecting less than 200,000 individuals in the US. Most importantly, the Orphan Drug Designation comes with some incentives like tax credits towards the clinical trial cost and prescription drug user fee waiver. Once a product with Orphan Drug Designation has received its first FDA approval for the indicated disease for the designation, it will be entitled up to 7 years of market exclusivity.
The company’s CEO and President Manuel Litchman said that the grant of Rare Paediatric Disease and Orphan Drug Designations for MB-2017 in treating XSCID patients that have previously received HSCT and requiring retreatment are huge milestones for the company. He added that5 they are an indication of the huge unmet medical need for such a rare disease.
Orphan Drug and Rare Paediatric designations huge milestones
Litchaman indicated that achievements bring the company closer to offering a new possible treatment alternative to patients and kin. Mustang is looking forward to commencing pivotal MB-207 clinical programs in previously transplanted XSCID patients and MB-107 in newly diagnosed XSCID infants.
The company co-developed the lentiviral gene-therapy with the National Institute of Allergy and Infectious diseases scientists and St. Jude Children’s Research Hospital. NIAID has been studying MB-207 since 2012 and continues to assess it in NIAID-supported phase 1/2 XSCID clinical study in children above two years who previously received HSCT. Mustang will file an investigational new drug application to commence a pivotal second phase MB-207 clinical trial in Q4 2020.