For the first time in 12 years, the FDA has ap­proved a new front­line treat­ment for the most com­mon form of liv­er can­cer.

The agency okayed a com­bi­na­tion of Roche’s an­ti-VEGF an­ti­body Avastin and their im­munother­a­py Tecen­triq for pa­tients with un­re­sectable or metasta­t­ic he­pa­to­cel­lu­lar car­ci­no­ma (HCC). The ap­proval comes two weeks af­ter Roche and their big biotech sub Genen­tech pub­lished Phase III re­sults show­ing the com­bo im­proved both pro­gres­sion-free sur­vival and, cru­cial­ly, helped pa­tients live longer than the long-run­ning stan­dard-of-care, Bay­er’s Nex­avar.

In that study, 84% of the 336 pa­tients tak­ing Avastin and Tecen­triq com­bo were alive at 6 months, com­pared to 67.2% of the 165-pa­tient Nex­avar arm. At 12 months, 72.2% of the Roche pa­tients were alive vs 54.6% for Nex­avar.

Richard Finn

“For the first-time we have a reg­i­men that marked­ly im­proves sur­vival over so­rafenib, the stan­dard of care for first-line he­pa­to­cel­lu­lar car­ci­no­ma since 2007, and of­fers pa­tients the op­por­tu­ni­ty for im­proved dis­ease con­trol with a fa­vor­able tol­er­a­bil­i­ty pro­file,” lead in­ves­ti­ga­tor Richard Finn said in a state­ment, us­ing the chem­i­cal name for Nex­avar.

The ap­proval is a ma­jor boon for Roche’s im­munother­a­py pro­gram, which, while pro­duc­ing a block­buster, has nonethe­less long lagged be­hind the mar­ket-lead­ing drugs from Bris­tol My­ers Squibb and Mer­ck. No­tably, Bris­tol My­ers’ Op­di­vo failed last year to give pa­tients sta­tis­ti­cal­ly sig­nif­i­cant im­proved sur­vival over Nex­avar in the same set­ting, al­though that tri­al was as a monother­a­py and the NJ phar­ma in­di­cat­ed they may try to pur­sue oth­er front­line us­es for the drug in HCC.

The new in­di­ca­tion could al­so make a larg­er im­pact, both in rev­enue and for pa­tients, than last week, when Tecen­triq won ap­proval as a monother­a­py in non-small cell lung can­cer by show­ing it could beat chemother­a­py — years af­ter Mer­ck’s keytru­da had done the same. Roche has been oc­cu­py­ing a sim­i­lar po­si­tion in triple-neg­a­tive breast can­cer, af­ter it be­came the first im­munother­a­py ap­proved for that in­di­ca­tion last year.

For Roche, though, the big ques­tions now cen­ter on whether they can show that their an­ti-TIG­IT mol­e­cule tiragolum­ab — the first of the sec­ond-gen­er­a­tion check­point in­hibitor and an area where Roche is ahead of the pack — im­proves on ex­ist­ing ther­a­pies. So far, the da­ta have been mixed.

Pho­to: Richard Finn (Glob­al On­col­o­gy Acad­e­my)

Microcap biotech Seelos Therapeutics is halting enrollment of its study in spinocerebellar ataxia type 3 (also known as Machado-Joseph disease) because of “financial considerations,” and in order to focus on other studies, the company said today, adding that the pause would be temporary.

The study will continue with the patients who have already enrolled, and the data from them will be used to decide whether to continue enrolling others in the future.

The FDA today approved Emergent BioSolutions’ Narcan brand naloxone nasal spray for over-the-counter sales. The nod was expected and comes on the heels of a unanimous 19-0 advisory committee vote in favor of approval last month.

The move to OTC means the opioid overdose reversal agent will now be available on grocery, convenience and gas stations shelves, as well as potentially for purchase online.