Drugs Currently Being Tested in Clinical Trials

Find out:

  • what clinical trials are now recruiting in the UK  here.
  • what to do if you are interested in being considered for a clincial trial here.   
  • more about how clinical trials work here.

For an overview of progress, please see the Drug Pipeline.

Some of the drugs currently being tested in clinical trials:


ZOLGENSMA / AVXS-101 uses harmless, genetically-engineered viruses to increase SMN protein levels. In July 2016, it was granted ‘breakthrough status’ in the US to help speed up development. On 18th October 2018, AveXis submitted their regulatory applications for the treatment in the U.S., Europe and Japan for use in infants with SMA Type 1. In October 2018 NICE started its scoping work to see whether this treatment is a ‘correct topic’ they should look at.

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RG7916 / risdiplam (Roche) 

RG7916 / risdiplam, developed by Roche, is a small molecule that targets and encourages the SMN2 ‘back-up’ gene to produce a greater amount of functional SMN protein, which is lacking in people with SMA. In January 2017, the US Food and Drug Administration (FDA) granted RG7916 / risdiplam Orphan Drug Status.

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LMI070 / Branaplam (Novartis)

Similar to RG7916, LMI070 is a small molecule drug that increases the amount of SMN protein made by the SMN2 gene.

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Olesoxime (Trophos / Roche) 

Olesoxime is a neuro-protective drug that supports the nervous system under stressful conditions. In January 2015, a Phase II clinical trial with young people aged 3 – 25 with SMA Type 2 or 3, indicated that Olesoxime is safe, well tolerated, and able to positively impact disease progression. At the same time, Roche pharmaceutical company agreed to purchase the small, late-stage clinical drug testing company, Trophos that originally developed the drug.

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CK-2127107 / reldesemtiv (Cytokinetics)

CK-2127107 / reldesemtiv is a drug able to increase the force generated by skeletal muscle and delay the onset and extent of muscle fatigue. Cytokinetics, the pharmaceutical company developing CK-2127107 / reldesemtiv, recently announced that it is starting to enrol individuals with SMA Types 2, 3 or 4 in their phase 2 clinical trial in North America, where the drug was granted Orphan Drug status in May 2017.


SRK-015 (Scholar Rock)

SRK-015 is a muscle-directed therapy that aims to reverse or restrict the muscle atrophy and weakness experienced by SMA patients. The drug is an antibody that very specifically targets an important protein called myostatin.

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