Orphan drug status is designed to encourage the development of drugs that are needed but would be prohibitively expensive or unprofitable for drug companies to develop. The designation will give Sequella market-exclusivity rights and a faster, less costly path to approval, according to the Post.
The company hopes that SQ109 will reduce treatment time and the number of drugs required in standard regimens, the Post reports. Orphan drug status is a "big deal" for Sequella, Carol Nacy, the company's CEO, said, adding that it "really makes it a nice working relationship with the FDA, and we always like having good working relations with regulatory agencies".
SQ109 employs a mechanism different to other antibiotics used to treat TB. The experimental drug hinders cell wall synthesis and is active against multiple cellular pathways in a specific group of microorganisms, including TB bacteria.
The drug has shown strong in vitro activity against drug-susceptible and drug-resistant TB, including extensively drug-resistant TB, which is resistant to the two most potent first-line treatments and some of the available second-line drugs. It has also shown strong in vivo activity against pulmonary TB alone and in combination with other drugs.
Source: Kaiser Family Foundation