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Background Analysis: US FDA Advisory Committees to Discuss Objectives for a Clinical Development Program Aimed at Treating Achondroplasia – MAY 11, 2018 (PEDAC/EMDAC)


The US FDA has scheduled a joint meeting of the Pediatric Advisory Committee (PEDAC) and the Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) on Friday, May 11, 2018 to discuss drug development for the treatment of children with achondroplasia. The following topics should be considered for discussion: evidence required to establish dose-response, study design, study duration, intended population, and endpoints. In the open session, the committee does not intend to discuss any individual research programs. From 8:00 am to 11:00 am, the meeting will be closed to permit discussion and review of trade secret and/or confidential commercial information (5 U.S.C. 552b(c)(4)). During this session, the committees will discuss the premarketing drug development program of an investigational product.

Originally, this meeting was scheduled for March 22, 2018, but it was postponed due to inclement weather.

Indication Background

Description of Indication

Achondroplasia (ACH) is a form of short-limbed dwarfism. Although the word achondroplasia literally means "without cartilage formation," children with ACH do not have a problem with forming cartilage, but rather with converting cartilage to bone, particularly the long bones of the arms and legs. ACH is similar to another skeletal disorder called hypochondroplasia, but the features of achondroplasia tend to be more severe.

There are several health problems that are commonly associated with achondroplasia, including apnea (episodes where breathing slows or stops), obesity, and ear infections. Children with achondroplasia usually develop lordosis (a pronounced sway of the lower back) and bowed legs. Some affected people also develop kyphosis, an abnormal front-to-back curvature of the spine and back pain. More serious problems include spinal stenosis, a narrowing of the spinal canal compressing the spinal cord and hydrocephalus, a buildup of fluid in the brain.

The estimated frequency of achondroplasia has ranged from about one in 15,000 to one in 40,000 births.

Achondroplasia is caused by mutations in the fibroblast growth factor receptor 3 (FGFR3) gene. This gene contains instructions for making a protein that is involved in the development and maintenance of bone and brain tissue. Two specific mutations in the FGFR3 gene are responsible for almost all cases of achondroplasia.

Investigational Drugs for Achondroplasia

Companies with drug development programs for investigational ACH treatments include BioMarin Pharmaceuticals (Drug name vosoritide) and Therachon AG (Drug name TA-46). Vosoritide has orphan drug status in the US and European Union (EU). In the US, BioMarin has completed one Phase 2 study, has another active Phase 2 study, along with enrolling subjects in two Phase 3 studies. In February 2018, Therachon AG announced the beginning of a Phase 1 clinical trial, to take place in the Netherlands, with TA-46 for achondroplasia. TA-46 also has orphan drug status in the US and EU.

What’s Next?

Tarius will send a Briefing Summary after briefing materials are posted to FDA’s website (typically within 2 days of the meeting). This report will provide a summary of the FDA briefing materials.

Tarius will send a Results Wire soon after the meeting. This report will include the voting outcomes, if applicable, and key outcomes of the discussion.

METADATA: Sponsor: none Drug Name: none Drug Class: none Indication: achondroplasia

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DISCLAIMER: The information in this document is for informational purposes only. The SAC Tracker Background Analysis contains information from publicly available sources, including FDA, sponsor, scientific, and clinical websites. Tarius A/S assumes no liability for any inaccurate or incomplete information, or for any actions taken in reliance thereon. © Tarius A/S. All rights reserved.