Beyond Achondroplasia

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BioCentury published the article “Competing for growth”

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BioCentury´s Senior writer Mike Leviten wrote a substantial article about the current medicines in research and development for achondroplasia. The article was published on the 7th April.

This competition between pharma companies is very positive since it stimulates industry to develop better medicines and at a faster pace. The investment in research and development of medicines is tremendous and the best possible outcomes for the companies in this race is when the company provides the most effective medicine in reducing achondroplasia associated medical complications, produces less side effects and substantially increases the quality of life of the patient and his/her family.

When a chef is creating a new recipe, the best product is discovered sometimes after countless attempts, but the chef will never achieve the goal without tasting what is being created. And in drug discovery, “tasting” is when pharma works in order to gather all efforts to know who the patient is and what the patient lives and needs.

The following excerpt is available on Biocentury page:

With little in the clinical pipeline for achondroplasia beyond BioMarin Pharmaceutical Inc.’s late-stage vosoritide, startups Therachon AG and BioClin Therapeutics Inc. are rethinking how to tackle the disease. By targeting FGFR3 directly, the two companies think they can block a signal vosoritide misses, and produce compounds that have better efficacy and require less frequent dosing.

The field appears poised for an uptick of activity, triggered by BioMarin’s positive Phase II data and recent publications that validate the FGFR3 biology underlying the disorder.

Achondroplasia is the most common form of short-limb dwarfism, and is caused by a single point mutation in FGFR3 that constitutively activates the receptor and inhibits bone growth. The treatment landscape involves either painful limb-lengthening surgery or, typically, a one-year course of human growth hormone, which has limited benefit.

BioMarin’s approach is to block a downstream mediator in the FGFR3 pathway that is responsible for chondrocyte growth. Its candidate, vosoritide, is a stable analog of the peptide agonist CNP that acts via the cell surface receptor NPR2 to inhibit a ras-driven cascade downstream of FGFR3. In December, BioMarin initiated a Phase III trial of vosoritide in patients aged 5-14.

Ascendis Pharma A/S is taking a similar approach with its TransCon CNP, a sustained-release prodrug of CNP. The company plans to submit an IND this year (apply for an “Investigational New Drug” at the FDA and EMA).

By contrast, Therachon and BioClin are targeting FGFR3 activity directly, which allows them to block chondrocyte proliferation as well as growth.”

3 Comments

  1. Where is the drug offered to patients?
    I have a son aged 9.

    • There is currently no drug available for achondroplasia. BioMarin´s Vosoritide is the only one in clinical trials, phase 3.

  2. Dear researcher,

    I am Anna, and I am from Barcelona. My nephew is 5 months of age and has achondroplasia, as well. I knew about the study by BioMarin and would like him to enter the study. However, I was told that he was too young. I would like to know your opinion in regards to the possibilities for him to benefit from the effects of this drug within the subsequent years…

    Thank You for your response.

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