Roche and AstraZeneca snag 'breakthrough' tags for a pair of blockbusters in waiting

Roche ($RHHBY) and AstraZeneca ($AZN) picked up FDA breakthrough therapy designations for their top pipeline assets, moving forward in multiple sclerosis and cancer with treatments tabbed as potential blockbusters.

The agency bestowed its breakthrough tag on Roche's ocrelizumab, an injected therapy for multiple sclerosis that has turned heads with positive data in tough-to-treat patients. The FDA's move is based on Phase III data in which Roche's treatment reduced the risk of disability progression by 24% in patients with primary progressive MS, a particularly debilitating form of the disease that has long bedeviled drug developers. Ocrelizumab has also demonstrated strong results in the more common relapsing form of MS, and Roche has said it's on track to submit the treatment for FDA approval in the first half of this year.

Separately, AstraZeneca won a breakthrough designation for durvalumab, an immuno-oncology therapy designed to expose cancer to the body's natural defenses by blocking a protein called PD-L1. The FDA's move is based on early-stage data showing durvalumab can make a difference in advanced, drug-resistant urothelial bladder cancer, a devastating disease with 5-year survival rates under 15%, according to AstraZeneca. The company is pressing through a wide range of studies to establish durvalumab's efficacy in a host of cancers, hoping to compete with similar therapies from Bristol-Myers Squibb ($BMY), Merck ($MRK) and Roche.

The FDA's breakthrough program promises drugmakers preferred access to top agency officials throughout the development process, an effort to speed up the journey for promising medicines that might change the standard of care in serious diseases.

For Roche, ocrelizumab is a potentially disruptive force on the MS market, now dominated by oral therapies for patients with less severe disease and taxing infusions for more serious cases. In its clinical results so far, ocrelizumab has established itself as a potential Goldilocks treatment, besting the efficacy of pills like Biogen's ($BIIB) Tecfidera and Novartis' ($NVS) Gilenya while proving more tolerable than intravenous therapies like Sanofi's ($SNY) Lemtrada or Biogen's Tysabri. The treatment has the potential to bring in $2.7 billion a year for Roche by 2020, according to EvaluatePharma's consensus of analyst estimates.

AstraZeneca may face a rockier path with its high-profile therapy. In December, the company disclosed that an ongoing study pitting durvalumab against lung cancer--the treatment's lead indication--was unlikely to generate the data necessary to support FDA approval as a monotherapy. AstraZeneca has projected peak sales of $6.5 billion for durvalumab, but the changing landscape for so-called checkpoint inhibitors could put that goal out of reach.

- read Roche's statement
- here's AstraZeneca's release

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