Rare diseases specialist Swedish Orphan Biovitrum (Sobi) agreed to buy the drug emapalumab and is reorganizing to increase focus on late-stage development in hematology and immunology.
Catalyst Pharmaceuticals Inc., under fire for a high price tag on a rare disease drug, sued the U.S. Food and Drug Administration to challenge the recent approval of a lower-cost rival medicine.
Pharma companies may not like their products being the subject of ICER reports, but they can provide a jumping-off point for manufacturers to expand the conversation of the value of new medicines in the rare disease area.
The first rule of rare: There are no rules. There are over 7,000 different rare diseases – each with its own symptomology, history, and clinical and political landscape. Most are virgin territory for marketers; only 5 percent have an approved treatment.
According to the National Organization for Rare Diseases 2019 Fact Sheet, more than 90 percent of the 7,000 known rare diseases do not have an FDA-approved treatment. It’s a privilege to bring brands to market for the patients, caregivers, families and physicians who so badly need them. In order to effectively launch into the rare disease space, there are elements to consider that take us well beyond standard launch prep. It’s these elements that will set one brand apart from another and contribute to a successful launch.
The statistics are staggering and well-known. Despite an increasing pipeline of drugs targeting rare diseases, 95 percent of the 7,000 disorders classified as rare still have no indicated treatment option. Eighty percent of rare diseases are genetically based, and more than half of the 30 million Americans affected by rare disorders are children. Rare diseases can take five to seven years to be accurately diagnosed, partly because they are inherently difficult to identify and because there are relatively few physicians equipped to recognize and design an appropriate treatment plan for a disorder that will, at most, affect 200,000 people (but often far fewer).
Takeda Pharmaceutical Co. Ltd. will discontinue a late-stage study testing the company’s experimental treatment for amyloidosis, which did not meet the first of two main goals.
Cranbury, N.J.-based Amicus Therapeutics and the Perelman School of Medicine at the University of Pennsylvania are expanding their gene therapy collaboration.
Mallinckrodt plc announced that the company achieved target enrollment of 300 participants in the Phase 3 CONFIRM clinical study to evaluate the efficacy and safety of the investigational agent terlipressin in adult patients with Hepatorenal Syndrome Type 1 (HRS-1).
Pfizer announced plans to buy Therachon, a rare disease biotech company based in Basel, Switzerland.