Hello. I’m Jeff Stein, CEO of Trius Therapeutics. Welcome to the Trius Therapeutics CEO’s Blog, a new and exciting communication method that we will be using to reach our stakeholders. My subject today is the GAIN Act: Implications for Antibiotic Drug Developers

The anticipated ruling this week by the Supreme Court on the Affordable Care Act has overshadowed an important healthcare legislative action also expected this week – the Senate vote on the reauthorization of the Prescription Drug Users Fee Act, also known as PDUFA V.  Originally passed in 1992, PDUFA authorizes the FDA to collect fees from drug sponsors to expedite the review and approval of new medicines and has been cited as a key reason why the U.S. is considered a world leader in providing patients access to innovative drugs. PDUFA is one of the rare pieces of legislation that has broad support among the government, industry, academia and patients and, through timely reauthorizations, strengthens the U.S. healthcare system and facilitates access to potentially life saving new medicines.

An important category of life saving medicines highlighted in PDUFA V is antibiotics. Title VIII of PDUFA V, entitled “Generating Antibiotic Incentives Now” or the GAIN Act, contains provisions that provide industry with incentives to develop innovative antibiotics to treat life-threatening infections caused by drug resistant pathogens. Such antibiotics are called “Qualified Infectious Disease Products” or QIDPs under the GAIN Act.  Among the provisions of the Act, sponsors developing QIDPs may benefit from the following incentives:

1. Market exclusivity. QIDPs would be issued 5 years of market exclusivity in addition to the periods of exclusivity for which they would otherwise qualify. In the U.S., new small molecule drugs are granted 5 years of Hatch Waxman market exclusivity so this provision would provide a total of 10 years of market exclusivity to a sponsor with a new NDA for a QIDP.  What this means is that, for a period of 10 years from NDA approval of a QIDP, a potential competitor would not be able to use the data generated by a sponsor of the QIDP for the filing of an ANDA for a generic version of the sponsor’s antibiotic.

2. Priority review. Under the GAIN Act, NDAs for QIDPs would qualify for Priority Review by the FDA. As the FDA’s current goal for Priority Review is six months, this provision could cut in half the time to approval compared to the 12 month Standard Review goal most antibiotics are currently afforded.

3. Fast track review. Sponsors of QIDPs would be provided with early and frequent communications with the FDA, in addition to the typical review and communication opportunities.

4. Updated guidance. The GAIN Act provides for a specific timetable for the FDA to develop and issue draft and final guidance and provides sponsors with the opportunity to request written recommendations from the Secretary of HHS on the guidance for antibiotic trials if such guidance is lacking.

5. Pathogen focused drug development. Requires the FDA to issue guidance on pathogen focused antibacterial drug development.

While all of the GAIN Act provisions provide advantages for antibiotic drug developers, Trius can potentially benefit most in the near term from provisions 2, 3 and 5. The Market Exclusivity provision would most benefit sponsors with QIDPs that have less than 10 years of patent exclusivity at the time of NDA approval. Since Trius is targeting the third quarter of 2014 for the potential approval of Tedizolid phosphate this could provide Trius with market exclusivity through the third quarter of 2024. However, Tedizolid phosphate has already been granted patent protection in the U.S. through 2028 and therefore does not require the Market Exclusivity provision of the GAIN Act.

By contrast, Trius could potentially benefit from the Fast Track and Priority Review provisions. One of the biggest challenges in the design of clinical trials is the cumbersome process by which sponsors must submit questions to the FDA and the length of time to receive a response to those questions. The Fast Track provision is designed to expedite this process and I believe that Trius could experience an immediate benefit once the GAIN Act is signed into law. Also, the opportunity to receive Priority Review for Tedizolid phosphate’s NDA, and the subsequent potential for FDA approval and launch by up to six months earlier than currently expected, could represent a major benefit.

I believe that the GAIN Act provision that has the biggest potential to foster the development of new antibiotics is provision 5 above – Pathogen Focused Drug Development. However, this will be part of a larger story I will address in my next blog on why there is a widely perceived unmet need for innovative antibiotics. The urgent need for such drugs catalyzed rare bipartisan support in both the House of Representatives, which approved the GAIN Act last week, as well as the Senate, which is expected to approve it this week. President Obama has targeted signing PDUFA V by July 4th.  Antibiotic drug developers such as Trius Therapeutics are waiting to celebrate and take advantage of this important new legislation.