May 24 is go­ing to go down as a big day in No­var­tis’ 2019 high­light reel.

Not on­ly did the FDA stamp an OK on the prospec­tive gene ther­a­py block­buster Zol­gens­ma, with a record-set­ting $2.1 mil­lion price tag, reg­u­la­tors al­so green-light­ed alpelis­ib, one of their oth­er top block­buster can­di­dates of the year.

Vas Narasimhan Linkedin

These are 2 of the slate of 4 block­busters the phar­ma gi­ant $NVS high­light­ed for a com­mer­cial roll­out this year. And it’s the third big ap­proval this year, be­hind the MS drug Mayzent, mak­ing 2019 a wa­ter­shed year for the com­pa­ny and new CEO Vas Narasimhan.

Alpelis­ib, ini­tial­ly code-named BYL719, will now hit the mar­ket as Piqray. It will be sold for HR+/HER2- ad­vanced breast can­cer with a PIK3CA mu­ta­tion — the first such tar­get­ed ther­a­py OK’d by the agency.

The da­ta cleared all the hur­dles need­ed for the FDA. Piqray plus ful­ves­trant near­ly dou­bled me­di­an pro­gres­sion-free sur­vival com­pared to ful­ves­trant alone in HR+/HER2- ad­vanced breast can­cer pa­tients with a PIK3CA mu­ta­tion (me­di­an PFS 11.0 months vs 5.7 months; HR=0.65). The ORR for the com­bo was 35.7% vs 16.2% for ful­ves­trant alone, p=0.0002.

It won’t come cheap. The drug will be sold at a whole­sale ac­qui­si­tion price of $15,500 a month, or right around $200,000 a year.

That’s a win­ner. It’s al­so a source of pride for No­var­tis R&D. The multi­na­tion­al bought Zol­gens­ma a year ago when it was well on its way, but they brought this drug all the way up from their dis­cov­ery group at NI­BR, mak­ing it a home­grown suc­cess sto­ry. And the FDA ex­e­cut­ed the ap­proval as the first un­der its re­al-time re­view pro­gram, which lets reg­u­la­tors start con­sid­er­ing an ap­proval be­fore the for­mal sub­mis­sion of the ap­pli­ca­tion.

More amaz­ing news. Thrilled to share this mile­stone ap­proval of our new tar­get­ed ther­a­py. More than ten years in the mak­ing, this pro­gram was sup­port­ed by count­less in­ter­nal NI­BR cham­pi­ons. Con­grats to all who car­ried this ther­a­py through to pa­tients. https://t.co/fb­Jt­DRqITx

— Jay Brad­ner, M.D. (@jay­brad­ner) May 24, 2019

“To­day’s ap­proval is ex­pect­ed to change the way we prac­tice med­i­cine in ad­vanced breast can­cer. For the first time, physi­cians can test for PIK3CA bio­mark­ers and de­vel­op a treat­ment plan based on the ge­nom­ic pro­file of a pa­tient’s can­cer,” said Fab­rice An­dré, a pro­fes­sor in the de­part­ment of med­ical on­col­o­gy at In­sti­tut Gus­tave Roussy in Ville­juif, France, and the lead in­ves­ti­ga­tor in the study.

No­var­tis is fresh off mak­ing some bold promis­es to an­a­lysts about the 25-plus prospec­tive drugs it has in the pipeline. That gives the still new CEO Narasimhan a sol­id foun­da­tion to build his com­pa­ny on new drug cam­paigns, with the in-house de­vel­op­ment work sup­ple­ment­ed by a slate of planned bolt-on deals.

At the end of January, the European Medicines Agency officially launched its new clinical trials info system (CTIS), although the migration to the new platform has only really just begun, and sponsors have until the end of January 2023 before all initial trial applications must be submitted through CTIS.

Overall, 56 clinical trial applications have been submitted in CTIS during the first 3 months since the launch of the system on Jan. 31, according to new data posted by the EMA. By comparison, about 4,000 new trials are authorized each year across Europe.

As concerns related to uptake and distribution continue to linger, Switzerland is among the first countries that plans to destroy hundreds of thousands of expired and unused Covid-19 vaccine doses.

The European country said it plans to destroy more than 600,000 doses of Moderna’s Spikevax Covid-19 vaccine as the doses have reached their expiration date.

However, Moderna CEO Stéphane Bancel told the World Economic Forum in Davos, Switzerland that he’s in the process of throwing 30 million doses in the garbage, exclaiming, “We have a big demand problem.”

More than 500 stakeholders sent comments to the FTC on whether the commission should look further into pharma middlemen, known as PBMs, with many of the commenters calling for more federal oversight.

Similar to the critical open comment period in a deadlocked FTC session last February, pharmacies and pharmacy groups are continuing to call out the lack of transparency among the top 3 PBMs, which control about 80% of the market.