FDA green and red lights: May 2024

Last month the US FDA approved Bristol Myers Squibb’s Car-T therapy Breyanzi for two additional uses, two months after the autologous treatment got another additional nod, and became the first Car-T therapy available for chronic lymphocytic leukaemia.

This took Breyanzi’s US-approved settings up to six. There was also good news for Amgen, whose Imdelltra became the first drug targeting DLL3 to gain the US regulator’s blessing, years after AbbVie’s rova-T crashed and burned; however, the going remained tough for China-originated anti-PD-(L)1 drugs, with camrelizumab scoring a complete response letter.

In total May saw the FDA hand out four oncology drug approvals, with accelerated approval for Lilly’s Retevmo in paediatric patients as young as two in three RET-positive cancers settings making up the balance. Camrelizumab, originated by Jiangsu HengRui but licensed to HLB Therapeutics, which is responsible for the US, received the only CRL.

The setback, specifically for a camrelizumab combo with the VEGFR-2 inhibitor rivoceranib/apatinib in first-line liver cancer, was due to manufacturing site defects, in addition to the FDA being unable to inspect some sites in Russia and Ukraine. 

Accelerated approvals

Amgen’s Imdelltra was approved on an accelerated basis for second-line SCLC on the back of the uncontrolled Dellphi-301 study, which impressed at last year’s ESMO conference, and the confirmatory phase 3 Dellphi-304 study – a key consideration for an accelerated nod – is under way, as are two other Imdelltra pivotal trials.

One of the two new indications for Breyanzi, for follicular lymphoma, was also given on an accelerated basis, while mantle cell lymphoma was granted full approval. Breyanzi’s green light in March for CLL, including the related small lymphocytic lymphoma, was also accelerated, based on the Transcend CLL 004 trial initiated by Breyanzi’s originator, Juno.

CLL was one of the first haematological cancers in which Car-T therapy was studied, but it ultimately didn’t end up being an early approved use. That fell to ALL and DLBCL, where Breyanzi, as well as Gilead’s Yescarta and Novartis’s Kymriah, are marketed – the first two in the important second-line setting.

Kymriah and Yescarta have a label in follicular lymphoma too, while the Yescarta version Gilead markets as Tecartus is approved in mantle cell lymphoma and adult ALL. However, no Car-T therapies beyond Breyanzi boast a CLL label.

Selected May 2024 US regulatory decisions in oncology

Note: AA=accelerated approval. Source: company statements.