CureDuchenne released the following statement from its founder and CEO, Debra Miller, following the decision by the FDA to send a Complete Response Letter to BioMarin for its exon-skipping drug drisapersen (Kyndrisa):
“We are disappointed that the FDA did not approve drisapersen, given the significant benefit that many Duchenne boys experienced when they were on an early and consistent treatment protocol of the drug. Duchenne is progressive and relentless muscle wasting disease, and we cannot lose time finding therapies.
“Our fight to cure Duchenne muscular dystrophy goes on. This disease, with its complex genetic roots, is unlikely to be cured by any single drug. Drisapersen was expected to be beneficial to about 13 percent of all patients.
“We appreciate BioMarin’s professionalism and dedication in developing this drug. And we are extremely grateful to the patients and families who participated in this demanding study over the last several years. We remain hopeful that drisapersen, after further research, can still become a useful tool against Duchenne, in Europe and potentially the U.S. BioMarin has other drugs in development for Duchenne which treat different mutations, and we are anxious to see the plans for those drugs go forward.”
Eteplirsen, developed by Sarepta Therapeutics, is another exon skipping drug which targets the same patient population. CureDuchenne’s early funding helped Sarepta get off of clinical hold and move into human clinical trials. CureDuchenne will be supporting Sarepta at its upcoming FDA Advisory Committee meeting on January 22, 2016. An additional CureDuchenne-funded company, PTC Therapeutics, has recently submitted a New Drug Application for FDA approval, and CureDuchenne looks forward to supporting PTC as it moves through the approval process.
BioMarin is reviewing the Complete Response Letter and says it will work with the FDA to determine the appropriate next steps for the drisapersen application.
Meanwhile, ongoing drisapersen extension studies will continue, as will the ongoing clinical trials for other exon-skipping compounds, known as BMN 044, BMN 045 and BMN 053. Patients currently receiving drisapersen, BMN 044, BMN 045 and BMN 053 will remain on therapy.
CureDuchenne is committed to treating the whole disease and has recently funded projects that will target cardiac failure in Duchenne patients – which is the primary cause of death – along with anti-inflammatory and anti-fibrotic drugs and gene therapy.
“To the Duchenne community, we pledge that we are in this for the long haul. We cannot rest until there are treatments – and, ultimately, cures – for all patients with Duchenne muscular dystrophy,” said Miller.
Duchenne muscular dystrophy (see an explanatory infographic here ) affects more than 300,000 patients, mostly boys and young men, worldwide. Because of a variant in one exon – a segment of genetic code in the cells of Duchenne patients – the body is unable to produce dystrophin, a protein critical to the formation of muscle tissue. The simplest of tasks become difficult for those with Duchenne, and in the later stages, heart and breathing muscles begin to fail. Boys with Duchenne are usually diagnosed by the age of 5 and lose their ability to walk by age 12. Most don’t survive their mid-20s.
Exon-skipping drugs, in effect, tell the body to ignore the damaged genetic material, thus encouraging the cells to produce new dystrophin and thereby maintain muscle strength.
Debra Miller and her husband Paul founded CureDuchenne in 2003 after their son, Hawken, was diagnosed with Duchenne. CureDuchenne has raised more than $20 million in the fight against Duchenne and has leveraged more than $100 million from biotech and pharmaceutical companies and other foundations to fund research leading to a cure.
CureDuchenne has used a successful venture philanthropy model, making investments and grants to support Duchenne research, and putting any proceeds into other promising pharmaceutical projects. In the last year it has invested $1 million in MyoTherix Inc., a biotechnology company focused on developing novel therapeutics for the treatment of Duchenne and other muscular dystrophies. It also has invested $1.5 million in Capricor Therapeutics, a biotechnology company, to advance promising research to treat heart muscle failure associated with Duchenne muscular dystrophy. CureDuchenne has already funded two research projects so far in 2016. A promising anti-inflammatory and ant-fibrotic treatment with RASRx and a gene therapy treatment with Bamboo Therapeutics.
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