InterMune ($ITMN) secured the FDA's breakthrough therapy designation for its in-development treatment of a rare lung disease, evening the regulatory score with rival Boehringer Ingelheim as the two angle for position on the U.S. market.
The biotech's drug, pirfenidone, treats the often-fatal idiopathic pulmonary fibrosis (IPF), a lung-scarring disease that inhibits oxygen absorption, leading to shortness of breath and poor pulmonary function. The FDA's breakthrough tag guarantees an expedited review and access to the agency's senior staff, but InterMune isn't expecting the nod to change the drug's timeline for approval.
Pirfenidone already picked up the FDA's promise of a priority review, and the addition of a breakthrough designation doesn't dial up the agency's speed, InterMune said in an email. That said, the FDA's promise to "involve senior FDA managers and provide additional resources to coordinate cross-disciplinary review" could make a difference in time, but that's entirely at the agency's discretion, InterMune said.
As it stands, InterMune expects to win approval for pirfenidone by November, launching the drug in the first quarter of 2015 if all goes according to plan.
The breakthrough designation comes just one day after Boehringer Ingelheim, maker of a rival IPF treatment, received the same honor. Like its competitor, Boehringer's nintedanib had already won a priority review designation, and the German drugmaker hasn't disclosed its expected timeline for approval.
InterMune's drug endured an FDA rejection back in 2010, but, after heading back to the clinic and returning with some stellar new Phase III data, the biotech worked its way back into the agency's good graces, resubmitting the drug in May.
There are no FDA-approved treatments for IPF, which kills about 40,000 people a year, according to the Coalition for Pulmonary Fibrosis. Pirfenidone has been on the market overseas for a few years but has yet to generate any notable sales momentum, bringing in just $70.2 million last year.
Analysts figure the U.S. market for IPF drugs could top out at above $2 billion, but whether InterMune or Boehringer claims the lion's share remains the subject of debate. Both drugs have charted impressive results in Phase III, but each has missed out on at least one secondary endpoint in its late-stage program.
Pirfenidone works by inhibiting two cytokines: TGF-beta, which controls a slew of cell functions and plays a part in fibrosis; and TNF-alpha, which has an active role in inflammation. In pivotal studies, the drug significantly reduced patients' decline in lung function compared to placebo, also meeting a secondary endpoint of performance on a 6-minute walking test.
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