PhRMA, ALS Association: more than 560 rare disease treatments in development

WASHINGTON — The Pharmaceutical Research and Manufacturers of America, in partnership with the Association, published a report Monday on the state of rare disease drugs currently in development. The report looks in-depth at the more than 560 drugs being developed that take new approaches to disease targeting. 

 

According to nonprofit Global Genes, some 30 million Americans live with a rare disease (defined as affecting fewer than 200,000 people), and only 5% of the approximately 7,000 known rare diseases have an approved treatment. 

 

But the trend in specialty pharmaceutical development is looking to produce treatments for these small patient populations, with the FDA approving 17 orphan drugs for rare diseases in 2015, making up 47% of novel new drug approvals last year. 

 

Among the treatments being developed are 151 for rare cancer and 82 for rare blood cancers, 148 for such genetic disorders as cystic fibrosis, 28 for such neurological disorders as ALS and seizures, 31 for infectious diseases and 25 for autoimmune disorders. 

 

“Biopharmaceutical researchers continue to persevere against numerous challenges in their quest to develop new treatments that can address patients’ unmet medical needs, specifically for those with rare diseases,” PhRMA president and CEO Stephen Ubl said. “While we are encouraged by the progress to date, it is critical to maintain an ecosystem that fosters and encourages the development of new medicines for the 30 million Americans currently living with a rare disease that still has few or no treatment options.”

 

The report includes the story of a patient who has lived with ALS for 12 years. The neurodegenerative disease has no known cure, but there are potential treatments currently under development. 

 

“It is collaborative efforts between biopharmaceutical researchers, patients and disease groups like ours that are driving the search for new treatments for ALS,” ALS Association president and CEO Barbara Newhouse, president and chief executive officer of The ALS Association. “As we learn more and more about this debilitating disease, the prospect of a cure is becoming a reality – giving patients and their families new hope.”

 

To view the full report, click here.