Commercializing Biotech
Biotechs sponsor a growing share of new drug approvals, and many are commercializing their products too. What drives a decision to “go it alone” – and what are the steps to success?
Biotechs sponsor a growing share of new drug approvals, and many are commercializing their products too. What drives a decision to "go it alone" – and what are the steps to success?
Biotechs were behind almost two-thirds of FDA’s 50-plus new drug approvals in 2024, and many are commercializing their products in the US alone. A decade ago, less than half of new drugs had biotech sponsors. Yet, then and now, plucky biotechs seek to sell their own products and to retain more of the fruits of their R&D.
Most fail. The exceptions – the likes of Gilead, Exelixis, Vertex, Alexion (now AstraZeneca) or, in Europe, Actelion (now Johnson & Johnson) – are just that. Tightening reimbursement and a fast-changing payer environment demands significant market access expertise and networking within US managed care. A stand-out drug for an unmet need is necessary, but not sufficient. Indeed, products that are too novel can be problematic too. Building out a new market, setting up novel diagnostic methods or changing a treatment paradigm requires resource, time and groundwork that smaller companies may lack. "Think twice, and twice more, before launching your own drug. It is unimaginably difficult and complex," warned one biotech CEO back in 2014.
That warning still holds. Yet some things have changed in biotechs’ favor.
Accelerating scientific progress and ballooning venture capital means more, better funded firms and more drug approvals. Consolidating Big Pharma now want mega-blockbusters to plug patent expiries and are less interested in orphan drugs; their outsourcing habit has broadened available contracting skills too. Experts are leaving streamlining corporates to join (or create) nimble start-ups, while precision medicine and genomics continue to slice up diseases and patient populations, multiplying the number of rare and ultra-rare conditions.
These trends have emboldened more biotechs to go it alone with their newly approved drugs, many of which address rare diseases. Ionis Pharmaceuticals’ R&D engine has already generated three US-approved products for partners; it’s marketing the latest, Tryngolza, on its own. (Tryngolza is the first treatment for a very rare metabolic disorder called familial chylomicronemia syndrome, which affects fewer than 1000 Americans). IntraBio and Zevra Pharmaceuticals are, similarly, selling their respective new treatments for the rare lysosomal storage disorder Niemann-Pick disease. "What is a partner going to do for us?" asks IntraBio CEO and president Mallory Factor. "We already work closely with the patients and patient advocacy groups."
A stand-out drug for an unmet need is necessary, but not sufficient.