ALS Drug Developer Cytokinetics Joins Rare Disease Day Efforts to Educate and Promote Change

Cytokinetics announced that it has joined with efforts by the European Organisation for Rare Diseases (EURORDIS) and the National Organization for Rare Disorders (NORD) to raise awareness for Rare Disease Day. Cytokinetics is focused on discovering, developing, and commercializing new types of muscle activators, and has developed the amyotrophic lateral sclerosis (ALS) drug candidate tirasemtiv.

Rare Disease Day was established in 2008 as an international campaign taking place every year on the last day of February. The campaign aims to increase public understanding of rare diseases. The day is observed in more than 80 countries, and is sponsored by NORD in the U.S. While a rare disease is defined as affecting less than 200,000 people, the mere quantity of rare diseases – amounting to 7,000 – makes the total health impact enormous. Today, it is estimated 25 million Americans live with a rare disease.

The theme this year was “Patient Voice,” emphasizing the fundamental role of the rare disease community in advocating the urgent needs of people living with such diseases. Patients, caregivers, and healthcare advocates play a crucial part in making legislators and regulators better aware of, and more actively working toward, fair access to life-changing therapies, care, and support.

“Patients with rare diseases, as well as their families and caregivers, face unique challenges, and deserve our support today and every day,” Robert I. Blum, president and chief executive officer of Cytokinetics, said in a press release. “We stand in solidarity with these patients, who inspire and motivate us as we collaborate with the ALS [amyotrophic lateral sclerosis] and SMA [spinal muscular atrophy] research and clinical communities to advance the development of new medicines.”

One such medicine, tirasemtiv, is currently in a Phase 3 clinical trial called VITALITY-ALS. The drug candidate is an activator of the fast skeletal muscle troponin complex and intends to increase the complex’s sensitivity to calcium — a crucial molecule for muscle contraction. This gives muscles extra strength while delaying fatigue. Tirasemtiv has been granted orphan drug designation and fast track status by the U.S. Food and Drug Administration (FDA), as well as orphan medicinal product designation by the European Medicines Agency for the treatment of ALS.

Antother drug, CK-2127107, which Cytokinetics developed in collaboration with Astellas as a potential treatment for SMA, is now in Phase 2 clinical trials.