Rare Disease & Gene Therapy — June 29, 2026
What to Watch
- BridgeBio’s Q3 2026 infigratinib filing — the live question is whether the FDA admits the body proportionality benefit into the label, a differentiator versus BioMarin’s Voxzogo and Ascendis’ Yuviwel that could reshape the achondroplasia market ahead of an early-to-mid 2027 launch.
- Epicrispr EPI-321 dose escalation — longer follow-up and higher-dose FSHD cohorts will test whether the early lean-muscle gains hold, and whether epigenetic DUX4 silencing can outpace the siRNA and RNA programs racing for the first approved FSHD therapy.
- 4FRONT-2 52-week readout (H2 2026) — the pivotal data point for 4D-150 in wet AMD and a likely valuation inflection for 4DMT’s platform licensing discussions, in a field thinned by setbacks at REGENXBIO and Adverum.
- AstraZeneca’s hypophosphatasia filing path — the missed adult HICKORY endpoint leaves the breadth of any efzimfotase alfa label open; timeline and endpoint-remediation strategy will shape whether the $3-5B forecast survives payer scrutiny against established Strensiq.
This brief highlights the edition’s top stories. Read the full June 29, 2026 edition → for all stories and analysis — or browse the Rare Disease & Gene Therapy archive.
