Cytokinetics receives FDA orphan drug status for Ck-2127107 to treat SMA


Biopharmaceutical company Cytokinetics’ CK-2127107has received orphan drug designation from the Office of Orphan Products Development of the US Food and Drug Administration (FDA) for the potential treatment of spinal muscular atrophy (SMA).

SMA is a severe neuromuscular disease affecting one person every 6,000 to 10,000 live births annually, which is regarded as a potentially fatal genetic disorder.

Cytokinetics is developing CK-2127107 in collaboration with Astellas Pharma.

Regarded as a next-generation, fast skeletal muscle troponin activator (FSTA), the drug is designed to treat chronic obstructive pulmonary disease (COPD) and certain other debilitating diseases or conditions associated with skeletal muscle weakness in addition to SMA.

CK-2127107 decelerates the rate of calcium release from the regulatory troponin complex of fast skeletal muscle fibres, thereby sensitising the sarcomere to calcium and allowing an increase in skeletal muscle contractility.

"We are exploring the potential of CK-2127107 to improve muscle function and physical performance in patients with SMA and we look forward to seeing results from our ongoing Phase 2 clinical trial later this year."

Cytokinetics executive vice president and research and development head Fady Malik said: “We are pleased that the FDA has granted orphan drug status to CK-2127107 for the potential treatment of patients with SMA, one of the most common potentially fatal genetic disorders due to progressive neuromuscular weakness, resulting in severe respiratory and ambulatory impairment.

“We are exploring the potential of CK-2127107 to improve muscle function and physical performance in patients with SMA and we look forward to seeing results from our ongoing Phase 2 clinical trial later this year.”

Orphan drug designation will provide the company with certain benefits and incentives, which includes a seven-year period of US marketing exclusivity from the date of marketing authorisation.

Astellas and Cytokinetics formed a collaboration in 2013 to advance novel therapies for medical conditions for muscle impairment and weakness.

In 2014, the companies expanded the collaboration to include certain neuromuscular indications such as SMA and to advance CK-2127107 into Phase II clinical development.

The collaboration agreement was further amended last year, giving Astellas exclusive rights to co-develop and commercialise CK-2127107 and other FSTAs in non-neuromuscular indications and certain neuromuscular indications, including SMA and amyotrophic lateral sclerosis, subject to specific Cytokinetics’ development and commercialisation rights.