More Than 560 Medicines Are in Development for Rare Diseases

New technologies, growing understanding of rare diseases provide hope for patients

America’s biopharmaceutical research companies are currently developing more than 560 medicines for patients with rare diseases, according to a report released by the Pharmaceutical Research and Manufacturers of America (PhRMA) and the ALS Association. Medicines in development include therapies for multiple myeloma, cystic fibrosis, amyotrophic lateral sclerosis (ALS), and enzyme-deficiency disorders, providing treatment options for patients who had few or no treatment options before.

Today, there are 7,000 known rare diseases, or diseases that affect fewer than 200,000 people in the United States. Given the relatively small number of patients each rare disease affects and the complexity of these diseases, the development of new treatments and cures is complicated. Despite these challenges, researchers are leveraging new technologies and growing scientific understanding of many rare diseases to develop groundbreaking therapies.

“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,” said Stephen J. Ubl, President and Chief Executive Officer of PhRMA. “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 new report, “Medicines in Development for Rare Diseases,” takes an in-depth look at the more than 560 orphan drugs, which utilize new approaches for targeting diseases. Examples of the medicines currently in development include:

  • 151 for rare cancers and 82 for rare blood cancers.
  • 148 for genetic disorders, including cystic fibrosis and spinal muscular atrophy.
  • 38 for neurological disorders, including ALS and seizures.
  • 31 for infectious diseases, including rare bacterial infections and hepatitis. 25 for autoimmune diseases, including systemic sclerosis and juvenile arthritis.

The neurological disorder ALS, also known as Lou Gehrig’s disease, is just one of the numerous rare diseases with no cure. New therapies under development, like antisense technology against SOD1, are a step toward helping patients and their families manage this disease.

“It is collaborative efforts between biopharmaceutical researchers, patients, and disease groups like ours that are driving the search for new treatments for ALS,” said 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.”

Source: PhRMA; May 9, 2016.