The US Food and Drug Administration (FDA)'s first Breakthrough Therapy Designations have been granted to two cystic fibrosis (CF) treatments from Vertex Pharmaceuticals.

One designation is awarded to Vertex' Kalydeco (ivacaftor) monotherapy, for potential additional indications beyond the drug's currently-approved use in CP patients aged six and over who have the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene; there are estimated to be around 2,000 such patients worldwide. The other is for a combination of Kalydeco with the investigational compound VX-809, based on Phase II combination data announced last year.

Multiple studies are currently underway to determine whether patients with other CFTR mutations may benefit from ivacaftor alone, and Vertex is also planning to start, shortly, a pivotal Phase III programme of VX-809 in combination with ivacaftor in people who have two copies of F508del, the most common CF mutation.

Breakthrough Therapy Designation was enacted as part of the 2012 Food and Drug Administration Safety and Innovation Act (FDASIA). The initiative is intended to expedite the development and review of a potential new medicine if it is "intended, alone or in combination with one or more other drugs, to treat a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development."

The first designations were welcomed by US Democratic Senator Michael Bennet who, with Republican Senators Orrin Hatch and Richard Burr, authored the Advancing Breakthrough Therapies for Patients Act, a bill included in FDASIA's drug approval and patient access component. The bill amends the Food, Drug, and Cosmetic Act to require the FDA to facilitate the development and expedite the review of breakthrough drugs.

The news is welcome for patients "who live their daily lives suffering from cystic fibrosis and are waiting to learn if potential new treatments can help them," said Sen Bennet. "Now, FDA will work with Vertex to move these potentially lifesaving treatments through the FDA's approval process quickly and safely - and hopefully they'll be in the hands of patients in the near future."

As he introduced the Act, Sen Bennet had pointed out that: "we're seeing major breakthroughs in drugs and other treatments for debilitating and terminal diseases, but we're not always getting them to patients through the most efficient and safe pathways." The inclusion of the "breakthrough" provision in FDASIA would provide the flexibility "to allow home-run treatments that show great promise early can reach patients more quickly. It also strikes a careful balance between providing regulatory certainty for developers of these breakthrough treatments and maintaining the level of drug safety and efficacy patients expect and deserve," he said.

For patients, the provision would allow the FDA the ability to move toward more innovative clinical trials, such as minimising the number of patients enrolled in trials with a placebo and shortening the duration of trials when scientifically appropriate, added the bill's sponsors.

"Our goal for the breakthrough designation is that it will help bring more and more lifesaving cures to patients more efficiently than ever before," says Sen Bennet.

Vertex says that that the implications of its two Breakthrough Therapy Designations cannot be determined at this time. The firm adds that it is working with the FDA and other global regulatory agencies to determine any potential implications of the designations to its ongoing and planned development activities, and subsequent regulatory submissions, for the two treatments.