Accelerated Approval

Every article we have published on Accelerated Approval, newest first.

THE DRUG DOCKET

A new class of myeloma drug was approved on a more sensitive test of remission

Zenbexus didn't have to outlive patients in a control arm to win approval — it had to clear their bone marrow of detectable disease faster. That shortcut is becoming the norm, not the exception.

3 min read

THE DRUG DOCKET

FDA clears first gene therapy for a rare disorder managed with round-the-clock cornstarch

GENGLYCOS treats the cause of glycogen storage disease type Ia, not just the symptoms cornstarch regimens manage — but it carries a long list of serious risks and an accelerated-approval asterisk.

3 min read

THE DRUG DOCKET

The first US hepatitis D drug comes with a boxed warning and an 8-year wait for proof

Hepcludex clears a bar most rare-disease drugs never reach: full U.S. approval. But FDA granted it on a surrogate marker, denied a bonus review voucher, and wants outcomes data that won't arrive until 2034.

4 min read

THE DRUG DOCKET

FDA clears sibeprenlimab for IgA nephropathy on proteinuria, not kidney function

Voyxact cut urinary protein by about half at nine months in the VISIONARY trial. Whether that slows kidney decline is the question the accelerated approval leaves open until 2027.

4 min read

THE DRUG DOCKET

A second oral HER2 lung-cancer drug clears FDA on tumour response, not survival

Hyrnuo (sevabertinib) is cleared for previously treated HER2-mutant NSCLC on response rate alone. The confirmatory trial that must justify it does not report until 2029.

4 min read

THE DRUG DOCKET

FDA clears the first drug for Barth syndrome, four months after rejecting it

Forzinity was approved under accelerated approval to improve muscle strength in patients weighing at least 30 kg. The confirmatory trial is not due to report until 2030.

4 min read