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BioMarin Pharmaceutical gets accelerated FDA approval to treat pediatric dwarfism

The U.S. Food and Drug Administration has granted San Rafael-based BioMarin Pharmaceutical accelerated approval to market a treatment that stimulates height growth in children diagnosed with a rare genetic bone disease, the pharmaceutical company announced Nov. 19.

The medication, called Voxzogo, is given as a daily injection to children age 5 or older who have been diagnosed with achondroplasia, also known as dwarfism. Achondroplasia occurs in 1 in 25,000 births.

The accelerated approval means the FDA considers Voxzogo a therapy that treats a serious condition and fills an unmet medical need, according to Jonathan Day, executive medical director, clinical science at BioMarin, adding the company has been developing the treatment for more than a decade. Voxzogo is expected to be available next month.

The FDA’s accelerated approval for Voxzogo was based on the outcome of a yearlong randomized, double-blind Phase 3 study that evaluated the efficacy and safety of the treatment, according to BioMarin. A double-blind study means neither the participants nor researchers know which treatment a specific individual is getting — the medication or a placebo.

BioMarin’s Phase 3, or pivotal, study enrolled 121 children with achondroplasia, Day said. After the year, 58 of the children that had been randomly assigned in the Voxzogo study enrolled in an open–label extension study, which is done to assess long-term tolerability of a new medication. Two years after they were given the drug, those who were not receiving the placebo were seen to have maintained improvement in height gain.

“That pivotal study was in children 5 to 14 years of age when they came into the trial,” Day said. “But they can continue to receive the drug up to 18 years of age, which is why the FDA has now approved the drug for use in children from age 5 until they stop growing.”

Achondroplasia affects more than short stature. The condition also creates functional problems because the rest of the body is disproportionate. People with dwarfism commonly have an enlarged head, short arms and legs. Complications can include sleep apnea and spinal stenosis, among others, with some requiring multiple surgeries, according to Day.

Ahmin Haider, a 13-year-old from the Bay Area, began participating in BioMarin’s clinical trials for the therapy when he was 5, according to his parents, Munira and Amer Haider.

“Ahmin was diagnosed at his 9-month well visit when I went to the pediatrician thinking of it to be a regular visit, except I came out with a major life-changing diagnosis,” Munira Haider said, adding there’s no family history for achondroplasia. “There was the initial grieving and shock just from the lack of knowledge, because we didn't know what it meant to have a child with achondroplasia.”

The family of five set out to learn how to manage the condition from a health perspective — the parents were trained to administer the daily injection to their son — and also how to help Ahmin’s emotional well-being.

The couple founded the Growing Stronger Research Fund to help with fundraising efforts aimed at supporting achondroplasia research.

“We applaud the FDA for recognizing the urgent unmet medical need for this progressive condition,” said Amer Haider. “As a parent of a child with achondroplasia, I see the availability of treatments that impact bone growth as an important step forward.”

Ahmin spoke briefly with the Business Journal about his journey so far.

“I feel good,” he said. “An injection every day is not exactly the most fun thing, but I know I have to do it, and if it can help to make my future life easier, more convenient, and maybe give me some more opportunities, then it’s worth it.”

Cheryl Sarfaty covers tourism, hospitality, health care and education. She previously worked for a Gannett daily newspaper in New Jersey and NJBIZ, the state’s business journal. Cheryl has freelanced for business journals in Sacramento, Silicon Valley, San Francisco and Lehigh Valley, Pennsylvania. She has a bachelor’s degree in journalism from California State University, Northridge. Reach her at cheryl.sarfaty@busjrnl.com or 707-521-4259.

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