The FDA gave full approval based on a Phase II study, without the need for Phase III confirmatory data, for Amylyx’s ALS drug Relyvrio but the $158,000 WAC could spark a debate over pricing.

Pfizer is buying Biohaven's migraine portfolio, but the rest of the pipeline is poised to be spun out and there have been two development setbacks. Despite verdiperstat's failure in ALS, the field had a same-day win with Amylyx's Relyvrio approval. 

The first gene-edited therapy will be submitted for sickle cell disease and transfusion-dependent beta thalassemia; it will compete with bluebird in both indications, but analysts anticipate blockbuster sales in excess of $2bn.

Deal Snapshot: The Dutch biotech planned to move its preclinical candidate, the EGFR-targeting LAVA-1223, into the clinic later this year. Seagen has shown interest in business development as Merck merger talks have quieted down.

While the Phase IIb SOLANO trial of ION449 met its primary endpoint, AstraZeneca had set a high bar of 70% LDL cholesterol reduction for competitiveness that the study did not hit.

SIGA chief scientific officer Dennis Hruby spoke with Scrip about how the company is developing its drug, Tpoxx, to address the monkeypox outbreak.