FDA OKs Tesaro’s ni­ra­parib for ovar­i­an can­cer, hand­ing the biotech a big win on the la­bel

The FDA has wast­ed no time in giv­ing Tesaro $TSRO a broad ap­proval for the use of its PARP drug ni­ra­parib, which will now be mar­ket­ed as Ze­ju­la as a main­te­nance ther­a­py for re­cur­rent ovar­i­an can­cer.

The OK and the la­bel that came with it rep­re­sents a set­back for Myr­i­ad Ge­net­ics $MYGN, which had ar­gued that its com­pan­ion di­ag­nos­tic would be need­ed to iden­ti­fy a spe­cif­ic group of ovar­i­an can­cer pa­tients with a bio­mark­er in­di­cat­ing that they would most like­ly ben­e­fit. But reg­u­la­tors blew past any di­ag­nos­tic qual­i­fi­ca­tion in giv­ing Tesaro the la­bel that it was look­ing for, vin­di­cat­ing CEO Lon­nie Moul­der’s vow that he could get an OK aimed at a large seg­ment of the mar­ket.

Tesaro had ar­gued that the ef­fi­ca­cy da­ta for ni­ra­parib qual­i­fied for an ap­proval for use in pa­tients who are ei­ther HRD pos­i­tive or HRD neg­a­tive. But they were op­posed by a part­ner who had a sol­id busi­ness case for ar­gu­ing that the drug should be re­served for pa­tients who would be most like­ly to ben­e­fit.

In the end, though, Tesaro is left with a big boast in its fa­vor: “Ze­ju­la is the on­ly PARP in­hibitor that has demon­strat­ed a clin­i­cal­ly mean­ing­ful in­crease in pro­gres­sion-free sur­vival (PFS) in women with re­cur­rent ovar­i­an can­cer, re­gard­less of BR­CA mu­ta­tion or bio­mark­er sta­tus.”

The OK al­so helps po­si­tion Tesaro against As­traZeneca, which just de­liv­ered stel­lar Phase III da­ta on its PARP Lyn­parza. Clo­vis, mean­while, gained an ap­proval for its PARP Rubra­ca late last year as a treat­ment for BR­CA-mu­tat­ed ovar­i­an can­cer that had proved re­sis­tant to at least two pri­or ther­a­pies.

It’s a trans­for­ma­tion­al mo­ment for Tesaro. Its drug was list­ed by Eval­u­atePhar­ma as one of the top drugs in the in­dus­try’s pipeline this year, with peak sales ex­pec­ta­tions hov­er­ing close to $2 bil­lion a year. And its stock climbed about 6% in af­ter-mar­ket trad­ing.

The ap­proval came months ahead of the FDA’s PDU­FA dead­line in June, in­di­cat­ing its in­ter­est and un­der­stand­ing of the field.

Mary Lynne Hed­ley, Tesaro

Ze­ju­la’s la­bel notes that it is in­di­cat­ed for the main­te­nance treat­ment of adult pa­tients with re­cur­rent ep­ithe­lial ovar­i­an, fal­lop­i­an tube, or pri­ma­ry peri­toneal can­cer who are in a com­plete or par­tial re­sponse to plat­inum-based chemother­a­py. And Tesaro im­me­di­ate­ly fol­lowed the news of the OK with plans to open up on the R&D pro­gram for the drug.

“Based on the un­prece­dent­ed re­sults of the NO­VA tri­al in women with re­cur­rent ovar­i­an can­cer, we pre­vi­ous­ly an­nounced the ex­pan­sion and re­fine­ment of our PRI­MA and QUADRA tri­als to in­clude a broad pa­tient pop­u­la­tion, and in the case of PRI­MA, elim­i­nat­ed the en­roll­ment re­quire­ment for a bio­mark­er se­lect­ed tu­mor. With the ap­proval of ZE­JU­LA in hand, we will now be­gin to ex­e­cute on our plans to pur­sue po­ten­tial­ly trans­for­ma­tion­al ap­pli­ca­tions of ni­ra­parib in a broad range of metasta­t­ic can­cer in­di­ca­tions,” said Mary Lynne Hed­ley, pres­i­dent and COO of TESARO. “We plan to ex­pand our first-line ovar­i­an can­cer strat­e­gy to in­clude a com­bi­na­tion study that as­sess­es the po­ten­tial ben­e­fit of ni­ra­parib plus an an­ti-PD-1 an­ti­body in the main­te­nance set­ting and ini­ti­ate a clin­i­cal study of ni­ra­parib in com­bi­na­tion with be­va­cizum­ab in pa­tients with a first re­cur­rence of ovar­i­an can­cer, with an in­tent to re­place chemother­a­py in this set­ting. We re­main strong­ly com­mit­ted to study­ing ni­ra­parib in the breast can­cer set­ting and al­so ex­pect to ini­ti­ate a new tri­al of ni­ra­parib in com­bi­na­tion with an an­ti-PD-1 an­ti­body in women with metasta­t­ic triple-neg­a­tive breast can­cer. Fi­nal­ly, our goal to move ni­ra­parib in­to in­di­ca­tions be­yond ovar­i­an and breast can­cers en­com­pass­es plans to ini­ti­ate a reg­is­tra­tion strat­e­gy for the first-line treat­ment of pa­tients with metasta­t­ic non-small cell lung can­cer that in­cludes a phase 2 tri­al of ni­ra­parib in com­bi­na­tion with an an­ti-PD-1 an­ti­body in pa­tients, re­gard­less of PDL-1 tu­mor ex­pres­sion, and a phase 3 tri­al of ni­ra­parib in com­bi­na­tion with an an­ti-PD-1 an­ti­body in pa­tients with high lev­els of PDL-1 tu­mor ex­pres­sion.”

5AM Ven­tures: Fu­el­ing the Next Gen­er­a­tion of In­no­va­tors

By RBC Capital Markets
With Andy Schwab, Co-Founder and Managing Partner at 5AM Ventures

Key Points

Prescription Digital Therapeutics, cell therapy technologies, and in silico medicines will be a vital part of future treatment modalities.
Unlocking the potential of the microbiome could be the missing link to better disease diagnosis.
Growing links between academia, industry, and venture capital are spinning out more innovative biotech companies.
Biotech is now seen by investors as a growth space as well as a safe haven, fuelling the recent IPO boom.

Biohaven CEO Vlad Coric (Photo Credit: Andrew Venditti)

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