Mustang Bio Inc. (NASDAQ:MBIO) announced that it has received Orphan Drug Designation from the FDA for its gene therapy MB-207 in treating X-linked combined immunodeficiency (XSCID), which also known as bubble boy diseases. The company is MB-207 evaluated in treating XSCID patients that have previously received hematopoietic stem cell transplantation (HSCT) whom retreatment is indicated.
Orphan Drug designation has benefits
In August 2020, the company was granted Rare Paediatric Disease Designation for the lentiviral gene therapy to treat XSCID patients who have previously received HSCT treatment. The grant of the Orphan Drug designation is for biologics and drugs expected to offer safe and effective treatment, identification and prevention, rare diseases or disorders that affect less than 200,000 Americans.
Interestingly, the designation offers incentives like tax credits for clinical trial costs as well as a waiver of prescription drug fees. Suppose a drug or biologic with the designation is approved for a disease holding such designation. In that case, it will enjoy market exclusivity for seven years, which does not include intellectual property protection.
Mustang conducting phase 1/2 clinical study for XSCID
The President and Chief Executive of the Company, Manuel Litchman stated that the reception of the Orphan Drug Designation and the Rare Paediatric Disease Designation for MB-207 in treatment of XSCID patients previously treated with HSCT and for whom retreatment has been indicated is a huge regulatory milestone for Musang in addressing the unmet need of this rare disease. Litchman said that the achievements bring the company closer to having a potential treatment alternative for patients and their kin. Mustang Bio is looking forward to commencing its pivotal MB-207 clinical programs in patients previously transplanted with XSCID and MB-107 in children that have been diagnosed with XSCID.
Currently, the company is conducting a phase 1/2 clinical study for XSCID in newly diagnosed children less than two years at St. Jude UCSF Benioff Children’s Hospital in San Francisco and Seattle Children’s Hospital. The company is evaluating MB-107 for which it submitted an IND in May.
