RARE Daily

FDA Grants Fast Track Designation to Two Experimental Rare Disease Therapies

April 14, 2020

The U.S. Food and Drug Administration granted Fast Track designation to two experimental therapies targeting rare diseases: Alnylam Pharmaceuticals’ treatment for polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults and LifeMax Laboratories’ treatment for Netherton Syndrome.

Photo: Larry Hsu, co-founder and CEO of LifeMax

Fast track designation is granted by FDA to a drug that is intended to treat a serious condition and has nonclinical or clinical data demonstrating the potential to address such unmet medical need. The benefit of fast track designation includes, but not limited to, opportunities for frequent interaction with the FDA, eligibility for priority review and rolling review.

Transthyretin (ATTR) amyloidosis is a rare, progressively debilitating, and fatal disease caused by misfolded TTR proteins that accumulate as amyloid deposits in multiple tissues including the nerves, heart, and gastrointestinal tract. There are two types of ATTR amyloidosis: hereditary ATTR (hATTR) amyloidosis and wild-type (wtATTR) amyloidosis. hATTR amyloidosis is an inherited disease resulting in intractable peripheral sensory-motor neuropathy, autonomic neuropathy, and/or cardiomyopathy. It is estimated to affect 50,000 people worldwide, can have a debilitating impact on a patient’s life and may lead to premature death within 4.7 years of diagnosis.

Vutrisiran is an experimental, subcutaneously administered RNAi therapeutic in development for the treatment of ATTR amyloidosis, which encompasses both hereditary (hATTR) and wild-type (wtATTR) amyloidosis. It is designed to target and silence specific messenger RNA, blocking the production of wild-type and mutant transthyretin (TTR) protein before it is made. It is currently being tested in two late-stage trials to determine its safety and efficacy. Vutrisiran has also been granted Orphan Drug designation in the United States and the European Union for the treatment of ATTR amyloidosis.

“After completing enrollment earlier this year, we look forward to sharing topline results of the HELIOS-A phase 3 study of vutrisiran in early 2021,” said Rena Denoncourt, vutrisiran program leader at Alnylam.

The FDA also granted Fast Track designation for LifeMax Laboratories’ experimental treatment LM-030 for the treatment of Netherton syndrome, a severe autosomal recessive disease due to mutations in the SPINK5 gene.

Netherton is characterized by congenital erythroderma, or skin inflammation, “bamboo hair,” and immune system abnormalities. It can be life-threatening in pediatrics due to an impaired skin barrier that leads to severe dehydration, hypernatremia, hypothermia, gross weight loss, and sepsis. Failure to thrive is common in childhood as a result of chronic erythroderma, persistent cutaneous infection, malnutrition, and metabolic disorders. The severity of the skin abnormality in older patients can fluctuate over time. Most Netherton Syndrome patients also have other immune system-related disorders such as food allergies and asthma.

LM-030 was licensed from Novartis and is currently in a phase 2/3 pivotal clinical trial for the treatment of Netherton syndrome. LM-030 previously received Orphan Drug designation from both the FDA and European Commission as well as Rare Pediatric Disease designation from the FDA.

“Netherton Syndrome is a serious and debilitating disease that is potentially life threatening, especially in infants. LM-030 is the only program in clinical development for Netherton and could potentially become the first approved targeted therapy. This fast track designation will help expedite its development and bring this much needed therapy to patients in a timely manner,” said Larry Hsu, co-founder and CEO of LifeMax.

Author: Rare Daily Staff

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