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Santhera Pharmaceuticals announces that the Office of Orphan Products Development (OOPD) at the US Food and Drug Admin-istration (FDA) has granted Santhera an award of USD 246,000 in support of its ongoing Phase I trial with omigapil (CALLISTO) in patients with congenital muscular dystrophy (CMD). Santhera is conducting CALLISTO in collaboration with the US National Institutes of Health (NIH). The FDA awards grants through the Orphan Products Grants Program to sup-port the clinical development of products for use in rare diseases where no current therapy exists.
”We are delighted that the FDA has awarded us this prestigious and highly competitive grant in support of our CALLISTO trial in CMD,” commented Thomas Meier, PhD, CEO of Santhera. “With this award the FDA emphasizes the need for a therapy for CMD and the contribution of the CAL-LISTO trial to the development of an effective treatment. After Raxone, omigapil is our second pipe-line product which further demonstrates our dedication to developing effective medicines for the treatment of mitochondrial and neuromuscular diseases.”
“As the leading organization representing the interests of patients with CMD, we are very pleased that the FDA recognizes the need to advance medical research in this orphan disease. With cur-rently no effective treatment available for patients, the CALLISTO trial offers hope to patients with this devastating disease,” added Patrick May, President of CureCMD.
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 prod-ucts. Santhera, in collaboration with the US National Institutes of Health (NIH), is currently conduct-ing CALLISTO under the leadership of Prof. Carsten Bönnemann and Dr. Reghan Foley at the Na-tional 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.
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.
