Rare diseases, gene and cell therapies, orphan drug access and high-cost therapy reimbursement.
The FDA has approved zilurgisertib (Atebrioz) for fibrodysplasia ossificans progressiva in patients aged 12 and older, making it the third approved FOP therapy and the second in five weeks.
The FDA approved rebisufligene etisparvovec (Fayuvi) on September 17, 2026 — the first therapy cleared to alter the course of Sanfilippo syndrome type A in pediatric patients, giving Ultragenyx its …
mAbxience and Sandoz have signed a global collaboration agreement to develop and commercialize a biosimilar candidate of emicizumab, targeting the haemophilia A market valued at $5.7 billion.
Anvisa cancelled Elevidys’s conditional registration on August 24 at Roche’s request, citing insufficient safety and efficacy data — ending Brazilian access to the country’s most expensive approved drug.
ANVISA approved BridgeBio’s acoramidis on May 6 for ATTR-CM in an all-comers indication, citing ATTRibute-CM data showing 42% reduction in all-cause mortality and cardiovascular hospitalizations at 30 months.
Biogen reports $2.5B Q1 revenue, +2% YoY, with non-GAAP EPS of $3.57, up 18%. LEQEMBI’s 78% 18-month persistence rate and a new high-dose SPINRAZA FDA approval signal commercial and pipeline …
Mendoza becomes the first Argentine province to screen newborns for spinal muscular atrophy at birth, enabling pre-symptomatic treatment under a public-private accord with Novartis effective May 24, 2026.
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