2021-11-21| Trials & Approvals

BioMarin’s Dwarfism Drug Garners FDA Nod Amidst Concerns Over Threat to Dwarf Pride

by Manju Bhaskar
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After notching an approval in Europe in August, BioMarin has snagged an FDA nod for its dwarfism drug, Voxzogo, for treating children five years of age and older with achondroplasia. The once-daily injection is approved under accelerated approval but on the condition of a post-marketing study that will assess final adult height.

Achondroplasia is the most common form of skeletal dysplasia leading to disproportionate short stature. It is characterized by slowing of endochondral bone growth and disordered architecture in the long bones, spine, face, and base of the skull.

It is caused by a gain of function mutation in the fibroblast growth factor receptor 3 gene (FGFR3), a negative regulator of bone growth. The condition occurs in about one in 25,000 live births.

Jean-Jacques Bienaimé, Chairman and CEO of BioMarin, said that Voxzogo is the first FDA-approved therapeutic treatment for achondroplasia targeting the underlying cause of the condition.

Lynda Polgreen, M.D., of UCLA, an investigator in clinical trials for Voxzogo, strongly believes this approval is an important milestone representing the first time that physicians will be able to offer a therapy targeted at the root cause of the condition for families of children with achondroplasia aged five and older.

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Benefits of Voxzogo in Achondroplasia

Voxzogo, a C-type natriuretic peptide (CNP) analog, represents a new class of therapy, which acts as a positive regulator of the signaling pathway downstream of FGFR3 to promote endochondral bone growth. It is indicated to increase linear growth in pediatric patients with achondroplasia five years of age and older with open epiphyses (growth plates).

The approval was based on the outcomes of a Phase 3 study evaluating the efficacy and safety of Voxzogo in 121 children with achondroplasia aged between 5 and 14.9 years. The baseline mean annualized growth velocity (AGV) in the placebo and Voxzogo groups was 4.06 cm/year and 4.26 cm/year, respectively.

At week 52, the change from baseline in AGV was -0.17 cm/year for the placebo-treated patients and 1.40 cm/year for the Voxzogo treated patients, which is a statistically significant improvement of 1.57 cm/year in favor of Voxzogo.

Voxzogo continues to be studied in a broad clinical development program in achondroplasia, and safety and efficacy are being further evaluated across different ages and over time. To date, 243 children with achondroplasia from eight countries have been enrolled in seven BioMarin clinical studies evaluating the drug.

Voxzogo is being studied in children whose growth plates are still “open,” typically those under 18 years of age. This is approximately 25% of people with achondroplasia.

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Availability in the US

BioMarin develops and commercializes innovative therapies for patients with serious and life-threatening rare genetic diseases. The company’s portfolio consists of seven commercialized products and multiple clinical and pre-clinical product candidates.

As a part of BioMarin’s Voxzogo development program, continued approval for this indication may be contingent upon the verification and description of clinical benefit in confirmatory studies and BioMarin’s intention to use the ongoing open-label extension studies.

Voxzogo is expected to be available in the US by mid-to-late December, and BioMarin will begin the promotion of Voxzogo immediately.


Ethical Quandary

“BioMarin continues to support the achondroplasia community and has a long track record of advancing the standard of care in rare genetic conditions,” said Amer Haider, Co-Founder of Growing Stronger, a nonprofit organization raising donations granted as funds for researchers focused on dwarfism.

However, the decision was met with criticism by some members of the achondroplasia community as they consider dwarfism as a difference and not a disability that must be treated.

Little People of America (LPA), the world’s oldest and largest support organization for people with dwarfism, believes its responsibility is to help members better understand patient rights and advocate for a research focus on healthcare outcomes beyond growth velocity.

“With the FDA approval of BioMarin’s Voxzogo therapy, the dwarfism community has been challenged with the impact of this new treatment on its health, culture, and standing within society. LPA strongly believes that a focus on growth velocity is a search for a pharmaceutical solution for a societal problem,” said LPA President Mark Povinelli in a statement.

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