FDA awards LAMA2-CMD therapy MDL-101 orphan drug status
Therapy was recently granted agency's rare pediatric disease designation
MDL-101, Modalis Therapeutics’ epigenetic editing therapy for LAMA2-related congenital muscular dystrophy (LAMA2-CMD), has been granted orphan drug status by the U.S. Food and Drug Administration (FDA).
The designation is intended to encourage the development of therapies for serious or life-threatening rare diseases, which are those affecting fewer than 200,000 people in the U.S. The designation provides certain incentives, such as fee waivers and seven years of market exclusivity, if the therapy is ultimately approved.
MDL-101 was recently granted the FDA’s rare pediatric disease designation, which is meant for potential treatments for rare diseases that primarily affect children and adolescents younger than 18.
“Currently, there is no approved treatment for LAMA2-CMD in the United States. We are hopeful that MDL-101, which has the potential to activate LAMA1, the sister gene of LAMA2, the causative gene of this disease, and achieve a fundamental cure, will become the first treatment that improves the prognosis of these patients,” Haru Morita, CEO and president of Modalis, said in a company press release.
What causes LAMA2-CMD?
LAMA2-CMD, or merosin-deficient CMD type 1A, is caused by mutations in the LAMA2 gene, which provides instructions to produce the alpha-two subunit of the laminin-2 protein. The mutations result in the complete absence of the alpha-two subunit, which has a key role in muscle fiber stability.
Symptoms of the disorder include muscle weakness, and children with it usually don’t develop the ability to walk and have speaking and breathing difficulties. Scoliosis, a sideways curvature of the spine, is also common.
No therapies have been developed so far to address the underlying disease cause and gene therapies aren’t feasible because the LAMA-2 gene is too large to fit into commonly used viral vectors.
MDL-101 uses CRISPR-based epigenetic editing technology packaged into a viral vector that targets muscle cells to increase the activity of the LAMA-1 gene, which produces a protein similar to the LAMA2 protein that’s lacking in these patients. The treatment, meant to be given as a single dose, is expected to compensate for the lack of LAMA2 and ease LAMA2-CMD symptoms.
CRISPR is a molecular tool that’s used to change or modify pieces of a cell’s DNA. Epigenetics refers to changing how genes are switched on and off, but not changing the DNA sequence itself. According to Modalis, its CRISPR-GNDM technology may confer sustained effects over years or decades.
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