Make that 2 prospective blockbuster approvals in 1 day for Novartis as FDA OKs targeted cancer therapy
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.
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.