BioMarin nabs FDA review for dwarfism drug vosoritide, forgoes AdComm as it eyes 2021 approval

The FDA will hand down a decision on BioMarin’s rare disease drug vosoritide by Aug. 21 as it says there's no need for an advisory committee.

The U.S. regulator has accepted BioMarin’s NDA for vosoritide, the biopharma’s investigational, once-daily injection analog of C-type natriuretic peptide (CNP) for children with achondroplasia, the most common form of disproportionate short stature, also known as dwarfism, in humans.

BioMarin says this is the first time an application has been accepted for a treatment for achondroplasia in the U.S.; the European Medicines Agency in Europe has already validated its marketing application.

Though this can change, the FDA told BioMarin that it is not “currently planning to hold an advisory committee meeting to discuss the application.”

There is a slight wrinkle: The FDA reminded the company that two years back it made a recommendation that these sorts of applications will need two-year controlled trials in different age groups, which it doesn’t have.

BioMarin thinks it can smooth this out with its “highly persuasive” outcomes from the one-year randomized, double-blind, placebo-controlled phase 3 trial when coupled with data from a midstage program that has up to five years of long-term follow-up that has been compared to natural history data on growth.

Its phase 3 study pitted vosoritide against placebo in 121 children aged 5 to 14 who have the genetic disorder and whose growth plates are still open.

Over one year, the treatment improved growth velocity from baseline by 1.6 cm after adjusting for placebo. The study also looked at a range of secondary endpoints including the proportionality of limbs to the body, joint geometry and quality of life measures.

Achondroplasia is caused by a mutation in the fibroblast growth factor receptor 3 gene. Short stature from the failure of cartilage to turn into bone is the most recognizable symptom, but people with achondroplasia can also face serious health issues such as sleep apnea or spinal stenosis, in which the narrowing of the spine puts pressure on spinal nerves. Some patients may need surgery to reduce pressure on those nerves or to straighten bowed legs.

"We are looking forward to working with the FDA and other regulatory authorities to evaluate the safety and efficacy of the potentially first pharmacological treatment for children with achondroplasia. In addition to the completed clinical studies included in the application, we have included a natural history study to understand the progression of achondroplasia and medical implications," said Hank Fuchs, M.D., president of worldwide research and development at BioMarin.

“Our extensive development program has been focused on addressing the underlying cause of achondroplasia. We continue to respect the community's range of views around treatment options. We are grateful to the families who have participated in the clinical trials, the advocacy groups from around the world, and the study investigators, all who have been on this journey with us to deliver a treatment choice and contribute to further understanding achondroplasia medically and scientifically.”