Zacharon Pharmaceuticals has announced a $210 million deal with drug powerhouse Pfizer to develop treatments for orphan diseases.
The small San Diego-based biotechnology firm will provide Pfizer with access to its drug identification technology to target treatments for rare diseases, including lysosomal storage disorders. These conditions can cause progressive deterioration in physical and mental state, and can also lead to early death.
Zacharon uses an unusual method of developing new drugs that focuses on glycans, which are carbohydrate chains that play a fundamental role in basic cellular activity.
The biotech firm gathered more than $2 million in grants following its presentation of data from preclinical cancer trials at the National Cancer Institute Investor Forum last November.
Big Pharma’s interest in orphan diseases is nothing new but this early-stage development deal with Pfizer represents a huge vote of confidence for Zacharon, since larger drug companies are usually hesitant to commit to a venture without proven results.
7,000 Orphan Diseases To Treat
Despite the increasing number of drugs on the market, orphan disease patients still go largely untreated. However in recent years, advances in research, support from governments and regulatory bodies and commercial ventures have all contributed to an increase in interest.
Experts estimate that anywhere between 5,700 and 7,000 orphan diseases currently exist, affecting over 620 million people throughout the world.
In modern markets most of these diseases are of genetic origin, while in developing nations they are also often caused by infection or exposure to toxic substances.
Key Players – Orphan Disease Drug Market
- Orphan Medical (US)
- Rare Therapeutics (US)
- Orphan Europe
- Orphan Pharma International (Europe)
- AOP Orphan Pharmaceuticals (Europe)
- Swedish Orphan
- Orphan Link (Japan)
- Orphan Australia
- Pfizer (US)
- Zacharon (US)