In approving three novel drugs last week, the FDA bought its annual haul to 43 – the highest number of new drug approvals for 19 years. The current total for 2015 stands just shy of the 45 new products approved by the FDA in 1996; the highest number on record since introduction of the Prescription Drug User Fee Act (PDUFA) four years earlier.
While the list of new drugs approved over the past 12 months (see below) shapes a broad narrative, encompassing both regulatory trends and the health of the pharmaceutical sector, it is perhaps fitting that two of the FDA's most recent approvals – Alexion Pharmaceuticals' Kanuma and Baxalta's Vonvendi – are both orphan drugs indicated for the treatment of rare diseases
Therapies granted orphan drug status by the FDA account for over a third of new drug approvals in 2015, and include some cancer treatments indicated for relatively small patient populations. Furthermore, the recent approval of Kanuma highlights another key trend of 2015; the rising value of priority review vouchers, which are awarded to manufacturers for successfully developing rare disease drugs. Alexion is now in possession of two vouchers, which could prove highly lucrative if the company chooses to sell them to another company.
The administration has maintained its aggressiveness in approving new cancer treatments more quickly than ever before. Just under a third of novel drugs approved by the FDA in 2015 to date are cancer therapies, and just three of these 13 products were not granted breakthrough designation or some form of expedited review process prior to approval.
The FDA has recently approved a number of cancer treatments – such asAstraZeneca's Tagrisso and Johnson & Johnson's Darzalex – months ahead of their designated PDUFA dates and set a tone for the year by granting accelerated approval for Pfizer's Ibrance on the strength of Phase II data. Helped by this regulatory speediness, but also championing its own R&D efforts, AstraZeneca recently noted that Tagrisso's progression from first clinical study to market occurred over a period of just 2 years and 8 months. Aside from new drugs, the FDA has also showed a willingness to grant rapid indication expansion for previously approved cancer therapies; Bristol-Myers Squibb's PD-1 inhibitor Opdivo representing the best case study.
New drug approvals in 2015 will also see competitive stakes particularly raised in some markets. The FDA has, for example, approved four new myeloma treatments over the course of the year, including three in the past month. Two of the most anticipated approvals this year were the PCSK9 inhibitors Praluent (Regeneron Pharmaceuticals/Sanofi) and Repatha (Amgen). Both reached the US market within a month of each other and offer comparable efficacy and safety at reducing high cholesterol in patients who cannot be adequately treated with statins; a boon for payers who have been able to play one drug off against the other as a means to secure cheaper pricing.
Both Praluent and Repatha are forecast to become blockbuster drugs at some point in the future and a healthy number of products likely to generate peak $1 billion-plus sales have been approved in 2015.
Johnson & Johnson's Darzalex continues to impress in clinical studies, while AstraZeneca's bullish expectations for Tagrisso appear to have been boosted by recent data disclosures from competitor Clovis Oncology .Having endured a two year-delay, Novo Nordisk secured long-awaited approval for its basal insulin Tresiba, while Pfizer's Ibrance has reportedly enjoyed a very strong launch.
From a corporate perspective, Novartis has enjoyed a productive year with the FDA, securing four novel approvals; more than any other company. Two of these products – Entresto and Cosentyx – are expected to generate substantial sales for the Swiss company. Alexion has trebled the number of products in its marketed portfolio by securing approval for Kanuma and Strensiq, marking a notable evolution in its growth model, which is built around treatments for rare diseases .
