bluebird bio started last week on tenterhooks, as FDA advisors cast their eyes over data for two gene therapy candidates that are key to its future as a commercially viable concern.
The FDA may have safety concerns abut bluebird bio's gene therapy for rare, fatal disease cerebral adrenoleukodystrophy (CALD), but its advisors believe its benefits far outweigh the risks.
Sanofi and Regeneron should hear from the FDA in the autumn whether it will approve their blockbuster immunology drug Dupixent as a therapy for rare skin disease prurigo nodularis (PN).
The UK regulatory authority has cleared the path for some patients with generalised myasthenia gravis (gMG) to get early access to Argenx' efgartigimod while it reviews the marketing applic
Sanofi's Genzyme unit has moved a step closer to securing EU approval for Xenpozyme – its enzyme replacement therapy for rare disease acid sphingomyelinase deficiency (ASMD) – after scoring
PTC Therapeutic's Upstaza gene therapy for patients with genetic disorder AADC deficiency has been recommended for approval in the EU, setting up another test of the commercial prospects f