Date: 2016-08-30
Type of information: Grant
Company: Santhera Pharmaceuticals (Switzerland)
Investors: FDA (USA)
Amount: $ 246,000
Funding type: grant
Planned used: This grant will support Santhera's ongoing Phase I trial with omigapil (CALLISTO) in patients with congenital muscular dystrophy (CMD). Santhera, in collaboration with the US National Institutes of Health (NIH), is currently conducting CALLISTO under the leadership of Prof. Carsten Bönnemann and Dr. Reghan Foley at the National Institute of Neurological Disorders and Stroke (NINDS) in Bethesda, Maryland. CALLISTO assesses the pharmacokinetics, safety and tolerability of omigapil in ambulatory and non-ambulatory children affected by either of two subtypes of CMD (COL6-RD or LAMA2-RD). The study is expected to be completed in the first half of 2017. The CALLISTO trial was previously supported by a public-private partnership including two patient organizations, the US-based Cure CMD and the Swiss Foundation for Research on Muscle Diseases, and EndoStem, an EU 7th Framework program. In May 2016, Santhera received Fast Track Designation from the FDA for omigapil for the treatment of CMD. Omigapil was previously granted Orphan Drug Designation for CMD in both the EU and the US.
Others: * On August 30, 2016, Santhera Pharmaceuticals announced that the Office of Orphan Products Development (OOPD) at the FDA has granted Santhera an award of $ 246,000 in support of its ongoing Phase I trial with omigapil (CALLISTO) in patients with congenital muscular dystrophy (CMD). The FDA awards grants through the Orphan Products Grants Program to support the clinical development of products for use in rare diseases where no current therapy exists. Orphan Products Grants are intended for clinical studies evaluating the safety and/or effectiveness of products that could either result in, or substantially contribute to, market approval of these products.
Therapeutic area: Rare diseases - Genetic diseases - Neuromuscular diseases
