FDA Grants Orphan Drug Designation to Cytokinetics Therapy CK-2127107 for Spinal Muscle Atrophy

FDA Grants Orphan Drug Designation to Cytokinetics Therapy CK-2127107 for Spinal Muscle Atrophy

The U.S. Food and Drug Administration (FDA) has granted Cytokinetics’ investigational spinal muscular atrophy (SMA) treatment CK-2127107 Orphan Drug Designation. That gives it seven years of U.S. marketing exclusivity, tax credits for clinical research and a waiver from FDA user fees.

CK-2127107, developed in partnership with Astellas Pharma, is a next-generation fast skeletal muscle troponin activator. The compound acts to slow the release of calcium inside muscle fibers. Calcium is a signaling factor in muscle, telling proteins to start or stop contracting a muscle. By lowering calcium levels, CK-2127107’s developers hope the mechanism will become more sensitive to calcium, improving contractile ability.

Researchers have shown that the drug causes SMA mice to improve, and the treatment is now in a Phase 2 trial (NCT02644668) in SMA. The trial — which will test two doses of the drug compared to placebo — recently started recruiting patients to the higher-dose group.

“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,” Fady I. Malik, executive vice-president and head of research and development at Cytokinetics, said in a press release.

“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,” added Malik.

CK-2127107 is also being developed to potentially treat other conditions. The San Francisco-based company will explore the drug in a Phase 1b study (NCT03065959), aiming to reduce muscle fatigue among elderly patients with movement problems. In addition, a Phase 2 study (NCT02662582) in patients with chronic obstructive pulmonary disease will investigate whether CK-2127107 can reduce exercise intolerance and improve physical capacity.


  1. Sergio Di Vincenzo says:

    I am a 57 year old male in Perth WA with SMA type 3

    how do I get on some of these trials. as we know with age SMA deteriorates and I feel like everyone is fiddling whilst the older SMA patients are burning.
    If any of the recent announcements from Spinrazza,Avexis or Cytokenitics has any benefit to type 3’s why can we just be allowed to try it. I would like to hear about real improvements with Type 3’s on any product.

    • Ashley says:


      I have used this website to find clinical trials. You may be able to get in on the Cytokenitics trial. My husband with type 3 was in the Phase 2 trial. They should be moving forward with a Phase 3 trial sometime in the future.

      Spinraza is currently available for all SMA types. Call your neurologist to have him/her submit a start form that they can download from Sprinraza.com. There is also a helpline you can call with questions on how to get started. You can find administering locations near you from the http://www.curesma.org website. You just need your neurologist to write you a prescription and any qualified doctor will assess you and hopefully give you the treatment. There are limited sites administering the drug right now but more will be opening soon.

      Hope this helps!

        • Ashley says:

          I’m sorry to hear you can’t get into any trials in the US. We still haven’t found out if my husband was on the actual drug or on the placebo. He is ambulatory but has trouble doing stairs or getting up from a seated position, especially on low chairs with no arm rests. He did the Cytokinetics | CK-2127107 trial – Phase 2. It was an oral drug that helps aid with muscle movement and strength. There really wasn’t that much of a noticeable difference. I did notice he could get up out of the car better and wasn’t so stiff after being seated for so long (we had a 6 hour car ride from Wisconsin to Missouri) to be in the trial. Other than that we didn’t notice a whole lot so my husband is hoping he was on the placebo wanting the actual drug to result in a more noticeable difference.
          We are currently in the process of getting approved to start Spinraza, the new FDA approved treatment for all SMA types. Is that available for you yet in Australia? If you do some research online it looks like that has the biggest promise in terms of seeing improvement at the moment. Gene therapy and other drugs are also coming down the pipeline so there will be more options within the next couple of years (hopefully sooner). If you have questions about Sprinraza, once my husband starts I can keep you updated.

  2. Sergio Di Vincenzo says:

    Hi Ashley
    Thank you for responding. I am in Perth Western Australia and I have been told by my neurologist that I can not get on to a trial in the USA. Can you tell me a little about your husbands condition pre trial and after receiving treatment?

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