Rare diseases
Regulators on both sides of the Atlantic are pushing for the withdrawal of the rare disease treatment that accounted for just 1% of Amgen’s 2025 revenue. Nevertheless, Amgen continues to defend the medicine, which was acquired in the $3.7 billion buyout of ChemoCentryx.
The FDA greenlit 26 novel therapies in the first half of 2026, including four for cancer and six for orphan indications. Meanwhile, AstraZeneca and Johnson and Johnson took home a combined 11 of the agency’s 79 total approvals, including supplemental nods.
Saol Therapeutics is the latest biotech to resubmit for approval of a drug rejected under former FDA Commissioner Marty Makary, following REGENXBIO and Replimune.
Even as FDA approvals for biologic therapies fell in the first half of 2026, regulatory experts are optimistic about a turnaround in the rare disease space after the departure of key leaders at the agency. Still, there will continue to be tension between science and politics.
Vertex Pharmaceuticals’ acquisition of Crinetics Pharmaceuticals is the largest pickup in the company’s history, according to analysts at BMO Capital Markets.
BridgeBio’s Attruby preserves kidney function in patients with transthyretin amyloidosis cardiomyopathy, an effect that is “distinct” from other drugs in this space, according to Jefferies.
Many of the FDA’s decisions this quarter involve applications that have previously been delayed, declined or outright rejected, including one for an mRNA vaccine that became the center of controversy earlier this year.
The FDA approved the expansion of Casgevy, which had previously been greenlit for patients 12 and up, into a younger pediatric population under the agency’s Commissioner’s National Priority Voucher program.
Ipsen is penning its second acquisition of the week, this time securing Memo Therapeutics and its midstage monoclonal antibody in a deal that could approach $800 million.
Sarepta Therapeutics is seeking to convert the accelerated approval of its therapeutic exon-skippers for Duchenne muscular dystrophy to full despite the drugs’ failure to improve motor function in a confirmatory trial.
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