InterMune治疗IPF药物获得FDA突破性药物认证

InterMune公司最近透露FDA已经确认授予其开发的治疗原发性肺纤维化（IPF）药物prifenidone突破性药物疗法，但并不会加快FDA对该药物的上市审核进程。原发性肺纤维化会导致肺部结构损伤，从而抑制肺部吸入氧气致使患者呼气困难，甚至会导致死亡。在FDA的先期审查中，pirfenidone已经获得了相关专家的高度认可，因而此次获得突破性药物认证可能并不会加速这一药物的审核。

按照原计划，InterMune公司希望今年11月pirfenidone能够获得FDA审核，并于2015年上市。prifenidone的这一认证也仅比竞争对手Boehringer Ingelheim公司的IPF药物nintedanib晚一天。不过后者并未透露nintedanib上市审批的具体计划。

详细英文报道：

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.