The U.S. Food and Drug Administration accepted Copenhagen, Denmark-based Orphazyme’s New Drug Application (NDA) for arimoclomol for the treatment of Niemann-Pick disease Type C.

Shares of Reata Pharmaceuticals plunged more than 33 percent after the company announced a potential delay in plans to seek approval of omaveloxolone, an investigational treatment of Friedreich’s ataxia (FA).

Alexion Pharmaceuticals shuttered the company’s oral small molecule Factor D inhibitor program for a rare kidney disease following disappointing clinical data.

Med Ad News spoke with Jennifer Brekke, principal and CEO of SCOUT Health, and Michael Treff, president of Code and Theory, about the formation of Code and Theory Health, a new business unit of the two agencies specifically focused on healthcare.

Digital-first creative agency Code and Theory has established Code and Theory Health – a new business unit dedicated to servicing brands and organizations within the healthcare industry – and added Scout Health, a 100-person agency focused on rare diseases, orphan drugs and specialty pharmaceuticals.

Ohio-based startup Forge Biologics closed a $40 million Series A financing round that will be used to expand manufacturing capabilities and support the clinical development of a gene therapy program for rare diseases, including the typically fatal Krabbe disease.

U.S. Food and Drug Administration PDUFA dates on the calendar for July include an NDA review for Jazz Pharmaceuticals’ experimental medicine for cataplexy.

The Cardiovascular and Renal Drugs Advisory Committee of the U.S. Food and Drug Administration voted to recommend approval for Mallinckrodt’s investigational agent terlipressin to treat adults with hepatorenal syndrome type 1 (HRS-1).

The pharmaceutical industry has set its sights on rare diseases with high unmet needs. However, as competition ramps up, how should pharma adapt its approach to succeed in this environment? Daniel Zaksas, PhD, Senior Vice President, Director of Scientific and Medical Affairs at Dudnyk, part of Fishawack Health, shares his learnings from a decade working in rare disease.

South San Francisco-based Annexon, focused on drugs for autoimmune and neurodegenerative disorders, closed on a $100 million Series C financing led by Redmile Group.