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PTC Therapeutics opts for conditional approach in Duchenne

Authored by Paul Foster

As part of Liftstream’s commitment to rare diseases, in the run up to Rare Disease Day 2013 we will be profiling 10 companies that are leading rare disease research. In the tenth in the series we look at US based PTC Therapeutics.

PTC Therapeutics is a privately owned developmental stage biotechnology company founded on 1998 by current Chairman and CEO Stuart W. Peltz, Ph.D. and Board member Allan Jacobson, Ph.D. PTC Therapeutics focus is the discovery, development and ultimately commercialisation of orally administered, proprietary small-molecule drugs that target post-transcriptional control processes. They are currently pursuing these activities in Oncology, Genetic and Infectious Diseases.

PTC Therapeutics has entered into collaborations with both Celgene and AstraZeneca to develop their Oncology pipeline. From the genetic perspective PTC Therapeutics working closely with Rare Disease specialists Genzyme. They also have further collaborations with heavy hitters such as Gilead, Merck and Pfizer on undisclosed projects. As with many rare disease companies PTC Therapeutics gain significant funding from charities and patient groups.

This is especially the case for Ataluren their lead product for the treatment of nonsense mutation Duchenne Muscular Dystrophy (nmDMD). Nonsense mutations are mutations in the DNA which prevents the Dystrophin protein being produced to its full, functional length. nmDMD affects approximately 13% of the DMD population. PTC Therapeutics has recently completed Phase IIb clinical trials for Ataluren in nmDMD. The trial was a large learning curve for PTC Therapeutics only the low dose combination (10mg, 10mg and 20mg/kg morning, noon and night) demonstrated significant effect on the six minute walking test (6MWT). Whilst these results were disappointing PTC Therapeutics have announced their intention to commence Phase III clinical trials by the end of Q1 2013 and are scheduled for completion by the end of 2014.

PTC Therapeutics have been granted Orphan Drug designation and Fast track approval by the FDA and announced in December 2012 that the EMA had validated its Marketing Authorisation Application (MAA) based on the Phase IIb data. PTC Therapeutics is hoping to gain conditional approval, potentially by the end of 2013, whilst the Phase III clinical trials are ongoing. Conditional approval is a relatively new mechanism, only available to Orphan Drugs in Europe which allows the whole patient population access to the drug whilst clinical trials are still ongoing. This could lead to the first ever approved treatment for DMD and will give hope to the 4500 patients living with this particular form of DMD in the US and Europe.

Liftstream is supporting Rare Disease Day 2013. In profiling this company we hope to bring greater awareness to rare diseases and the companies working in this area. Liftstream is sponsoring the 2013 Orphan Drug Congress and we look forward to having contact with you at these events or by contacting us via Linkedin or Twitter


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