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14, August 2020
Updated 18, August 2020

BioMarin announced that the European Medicines Agency (EMA) validated the Company's Marketing Authorization Application (MAA) for Vosoritide, an investigational, once-daily injection analog of C-type Natriuretic Peptide (CNP) for children with achondroplasia. (press release here). The MAA review will commence from today [1].

What comes next?

If this was a relay race, it would be the moment when the 3rd runner passes the baton to the 4th and last runner.

Now, EMA will review the clinical trial data to decide whether or not to grant marketing authorization for Vosoritide. If EMA gives a positive decision, another stage will start: Vosoritide will be granted with Market Access (MA).

What is Market Access?

This will ensure the drug can be marketed yet, this does not mean that real access will take place. To evaluate patients’ effective access to an orphan drug, four dimensions must be considered [2]:
1. Availability of the drug
2. Ease of supply from patients’ point of view
3. Level of reimbursement
4. Delay between MA and effective access to the drug

Marketing authorization granted to orphan drugs is only the first step, as medicines reach patients when reimbursement decisions are implemented by national health systems (this applies to non-orphan drugs too) [3]. Success in launching new orphan drugs implies the ability of the manufacturer/pharma company to achieve a level of the reimbursed price (the cost the drug will have to each payer, many times, national governments) and a level of market access that is commercially viable on the global market. Access to orphan drugs is challenging in many countries because the legal and policy frameworks may be absent, funding may be insufficient, and/or payers do not see the justification with the prices for these therapies [4].


Regarding the FDA, BioMarin remains on track to submit a New Drug Application (NDA) in the third quarter of 2020. Vosoritide has Orphan Drug designation from the FDA and the EMA.

"The acceptance of the vosoritide application for review by the EMA is the culmination of years of scientific and clinical research to bring the potential first pharmacological treatment option for children with achondroplasia," said Hank Fuchs, M.D., President Worldwide Research and Development at BioMarin.  "Throughout our journey, we have worked together with advocacy groups and physicians to address the underlying cause and associated complications of achondroplasia through a robust data set for regulators to evaluate the safety and efficacy of vosoritide. We are committed to providing children with achondroplasia and their families a treatment option and look forward to continuing working with the European regulatory authorities."

Melita Irving, Clinical Geneticist at Guy's and St Thomas' NHS Foundation Trust, London, UK and investigator for the vosoritide clinical program at the Evelina London Children's Hospital said:

"The extensive research conducted in Phase 2 and 3 clinical trials, along with a natural history study, has provided more scientific and medical knowledge around skeletal dysplasia, especially achondroplasia than we've ever had before. These clinical studies provide a much better understanding of how to treat the underlying conditions associated with achondroplasia more effectively and less invasively, and with no drug therapy options previously available, vosoritide has the potential to make a meaningful impact on the daily lives of these children."

Now, presenting real-world (and ideally national) data to the Institute/agency that is the payer of pharmaceutical products in each country, in order for the national agency to validate the value of the orphan drug, at a medical, socio-economic and quality of life points of view and accept to pay the company for the drug, is absolutely key in most of the countries to have, in fact, an orphan drug available for patients.

1. Press release PRNewswire
2. Bourdoncle M et al, Patient Access to orphan drugs in FranceOrphanet J Rare Dis. 2019; 14: 47.
3. Zamora, B., Maignen, F., O’Neill, P. et al. Comparing access to orphan medicinal products in EuropeOrphanet J Rare Dis 14, 95 (2019)
4. Luca F, Improving market access to rare disease therapies: A worldwide perspective with recommendations to the industry, SAGE journals, November 2, 2018 

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