Modalis Therapeutics receives FDA Orphan Drug designation

Modalis Therapeutics has been granted U.S. FDA’s grant of Orphan Drug Designation (ODD) for MDL-101, a cutting-edge treatment for congenital muscular dystrophy type 1a (LAMA2-CMD). The orphan designation will provide Modalis with benefits including tax credits, eligibility for grant funding, and a seven-year period of market exclusivity following FDA approval. These incentives, provided under the 1983 Orphan Drug Act, make it more feasible for companies to invest in developing treatments for rare diseases with small patient populations.

This news follows last month’s update that MDL-101 also received the FDA’s Rare Pediatric Disease Designation. Together, these two designations designations underscore the potential impact of MDL-101 as a first-of-its-kind therapy for LAMA2-CMD, which currently lacks FDA-approved treatment options. Modalis Therapeutics continues to actively work on bringing this therapy to clinical trials.

Modalis Therapeutics’ mission, “Every Life Deserves Attention,” drives their commitment to bring innovative therapies to those affected by rare diseases for which no cures currently exist. The FDA’s Orphan Drug Designation for MDL-101 highlights the importance of advancing treatment options for rare diseases and brings new hope to patients and families affected by LAMA2-CMD.

More information on the MDL-101 gene therapy can be found here.