EMA Committee Recommends Marketing Approval For BioMarin’s Vosoritide To Treat Children With Achondroplasia From Age 2 Until Growth Plates Close

BioMarin Pharmaceutical Inc. announced that the Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending marketing authorization for vosoritide, a once daily injection analog of C-type Natriuretic Peptide (CNP) to treat achondroplasia in children from the age of 2 until growth plates are closed, which occurs after puberty when children reach final adult height. Achondroplasia is the most common form of disproportionate short stature in humans. A final approval decision, typically consistent with the CHMP recommendation, is expected from the European Commission in Q3 2021. Vosoritide is potentially the first medicine to be approved to treat children with achondroplasia in Europe and would be marketed under the brand name Voxzogo (vosoritide).It is estimated that over 11,000 children across Europe, Middle East, and Africa are affected by achondroplasia and eligible for treatment with vosoritide, if approved by a health authority. Approximately a third of this population are in countries authorized to sell under the EMA license. BioMarin anticipates additional patient access through named patient sales based on an EMA approval in countries in the Middle East and Africa and expects additional market registrations to be facilitated by an anticipated EMA license. Also, the French National Agency for Medicines and Health Products Safety (ANSM) granted an Autorisation Temporaire d'Utilisation de cohorte (ATU cohort), or Temporary Authorization for Use to allow access and reimbursement of vosoritide to begin immediately under an authorized process. An ATU allows access to drugs not yet approved in France, when provided for rare diseases, with no alternative options, and when the benefit/risk is presumed positive."The positive opinion from the CHMP represents a significant step towards making vosoritide available as a treatment choice for families. The CHMP opinion reinforces the strength of the data and the clinical benefit to children as young as two years old. The temporary authorization granted by the French health authority is a response to the urgency to treat these children," said Hank Fuchs, M.D., president Worldwide Research and Development at BioMarin. "We are committed to the scientifically robust, and rigorous study of vosoritide to continue to demonstrate the safety and efficacy of this investigational therapy that addresses the root cause of achondroplasia. We are committed to providing information to treating physicians and families to determine if a treatment choice is right for them. We are grateful to the patient advocacy groups, our clinical investigators and the children and their families for their participation in this clinical programme.""This positive CHMP opinion represents a significant step toward delivering on the promise of the first pharmacological treatment option for children and families affected by achondroplasia. As a treating physician, I see an urgent demand from families for a treatment option that addresses bone growth," said Klaus Mohnike, Professor of Paediatrics at Magdeburg University Hospital in Germany and investigator for the vosoritide clinical program. "The depth and breadth of the data that supports this opinion builds upon a strong scientific foundation that will continue to increase our understanding of the potential impact of vosoritide on the medical complications that may result from achondroplasia." "Currently, there is a significant unmet need in achondroplasia. Further research is needed to address serious health complications beyond disproportionate short stature. People with achondroplasia can experience foramen magnum compression, sleep apnea, and spinal stenosis," said Carmen Alonso Alvarez, managing director of Fundacion ALPE Foundation. "We commend everyone involved in this effort to bring the potential first medicine to treat children with achondroplasia, which offers the possibility to expand treatment options beyond surgical intervention, address an unmet medical need, and advance the standard of care for our community."The CHMP based its opinion on the totality of data from the vosoritide clinical development program including the outcomes from the randomized, double-blind, placebo-controlled phase 3 study evaluating the efficacy and safety of vosoritide. The phase 3 Study was further supported by the ongoing long-term safety and efficacy from the phase 2 dose-finding study, which showed that growth rates have been sustained above participants' baseline rates and above the expected annualized growth velocity for untreated children with achondroplasia throughout the five year observation period for which data are currently available. No acceleration of bone age was observed, suggesting that vosoritide is not reducing the total duration of the growth period. The data package included results from an ongoing phase 2 randomized double-blind study in infants and young children, including extensive pharmacokinetic and biomarker data, as well as preliminary growth data from participants in the 2 to 5 year age cohort. Data in sentinel study participants showed a positive effect on growth following two years of vosoritide treatment in subjects aged 2 to 5 years. In addition, the data package included the phase 3 extension study and extensive natural history data.The CHMP is a scientific committee composed of representatives from the 27-member states of the EU, and Iceland, Norway and Liechtenstein. The committee reviews medical product applications on their scientific and clinical merit and provides advice to the European Commission (EC), which has the authority to approve medicines for the EU.Vosoritide, administered in approximately 38,000 injections, was generally well tolerated at all doses. The majority of adverse events (AEs) were mild and no serious adverse events (SAEs) were reported as study drug related. Across all doses, injection site reactions and hypotension were the most common drug-related AEs. All injection site reaction events were transient. AEs of hypotension were mild and transient with majority being asymptomatic and reported in the context of routine blood pressure measurements with minimal clinical impact. No new safety findings were observed. There were no AEs related to disproportionate bone growth or bone pathology. There has been no evidence of accelerated bone age (as assessed by radiologists blinded to the age of the subjects) or negative changes in bone mineral density.Achondroplasia, the most common form of skeletal dysplasia leading to disproportionate short stature in humans, is characterized by slowing of endochondral ossification, which results in disproportionate short stature and disordered architecture in the long bones, spine, face and base of the skull. This condition is caused by a change in the fibroblast growth factor receptor 3 gene (FGFR3), a negative regulator of bone growth. Beyond disproportionate short stature, people with achondroplasia can experience serious health complications, including foramen magnum compression, sleep apnea, bowed legs, mid-face hypoplasia, permanent sway of the lower back, spinal stenosis and recurrent ear infections. Some of these complications can result in the need for invasive surgeries such as spinal cord decompression and straightening of bowed legs. In addition, studies show increased mortality at every age.

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