1Q 2023 witnessed the failure of a fair share of biotech companies, reminding investors of the downside risks inherent in drug discovery and development.
However, several new drug approvals continued to highlight longer term growth prospects across the sector. Biotech investors cheered new FDA drug approvals for Travere Therapeutics’ (TVTX) Filspari (sparsentan) to reduce proteinuria in adults with primary IgAN, and Apellis Pharmaceuticals’ (APLS) Syfovre (pegcetacoplan) for the treatment of geographic atrophy secondary to age-related macular degeneration (AMD).
Both drugs will launch with a premium price and will serve patients with limited or no therapeutic options.
Also, Reata Pharmaceuticals (RETA) rallied ~200% upon FDA approval of omaveloxolone (Skyclarys) for the treatment of Friedreich’s Ataxia (FA).
There was controversy surrounding the approval given the unclear mechanism of action of the drug, concerns over potential safety risks, and debate around clinical benefit. FA is an ultra-rare indication with an estimated 5,000-6,000 patients in the US with no approved treatment options before Skyclarys.
FDA continues to show flexibility to allow new treatment options to come to market that serve small populations of severe patients with no other alternatives.