Tuesday 26 August 2008
Santhera receives orphan drug status approval for congenital muscular dystrophy drug
Swiss pharmaceutical company Santhera announced on 15 August 2008 that it had received orphan drug status approval for Omigapil.
This drug was approved by the FDA (US Food and Drug Administration) and EMEA (European Medicines Agency) and should make development of the drug to treat congenital muscular dystrophy (CMD) easier and more financially viable.
Contents:
- What is Omigapil and what will it be used for?
- What does this mean for people with congenital muscular dystrophy?
- What does 'orphan drug status' mean?
- Further information and links
What is Omigapil and what will it be used for?
The exact mechanism of action of Omigapil (also known as SNT317) is not fully understood. However, it is thought that it interacts with a protein called glyceraldehyde 3-phosphate dehydrogenase, which is involved in cell death. By interacting with this protein, Omigapil may protect muscle cells from dying.
Santhera claim that Omigapil has been successful in an animal model for congenital muscular dystrophy (CMD), and these results will be presented at the World Muscle Conference in Newcastle in September. In clinical trials they intend to test the potential of Omigapil to treat three different forms of CMD - Ullrich congenital muscular dystrophy, Bethlem myopathy and merosin-deficient congenital muscular dystrophy (MDC1A).
What does this mean for people with CMD?
Orphan drug status should make it quicker and easier for Santhera to bring Omigapil to clinical trial. If it proves to be an effective treatment, orphan drug status should also speed up the marketing process. They are hoping to start the first phase of clinical trial by the end of this year. Although the drug will be initially trialled on just three types of CMD, if it is successful there is potential for it to be used to treat other forms of muscular dystrophy.
What does 'orphan drug status' mean?
'Orphan drug status' is awarded to drugs intended to treat rare conditions that companies otherwise might not be able to develop for financial reasons. Drugs to treat conditions that affect less than 5 people per 10,000 are eligible. Developing and testing drugs is a very expensive process so this designation gives incentives to companies to pursue them, even if the market for that drug is small.
On the financial side, orphan drug status gives tax and fee reductions or exemptions. Most importantly, the company will also be allowed marketing exclusivity for an extended time. The patent on the drug or treatment is protected for 7-12 years depending on the country. On a more practical note, they also gain access to specialised scientific advice and assistance. The EMEA has also set up a centralised procedure of marketing authorisation to make it easier for companies to get orphan drugs on the market throughout the EU.
Orphan drugs generally follow the same regulatory development path as any other pharmaceutical treatment, however, they may be granted permission to test the drug on fewer patients since it may be more difficult to recruit a high number if the condition is rare.
The Orphan Drug Act was signed in the USA in 1983. Since then, more than 200 drugs and biological products for rare diseases have been brought to market. In contrast, the decade prior to 1983 saw fewer than ten such products come to market. The success of this initiative prompted other countries around the world to follow suit. An orphan drug policy was adopted by the EU in 2000.
Further information and links
Find out more about congenital muscular dystropy
The Muscular Dystrophy Campaign funds two projects researching CMD. One is led by Professor Francesco Muntoni at University College London and the second by Professor Kate Bushby at University of Newcastle Upon Tyne.
Find out more information about the Muscular Dystrophy Campaign's current research grants.
