Sequella Lead Drug Compound Receives Orphan Drug Status From FDA
FDA and the European Medicines Agency (EMEA) independently reviewed Sequella lead drug compound, SQ109, and both agencies granted SQ109 orphan drug status for the treatment of tuberculosis. SQ109 shows excellent in vitro and in vivo activity against drug-susceptible and drug-resistant TB bacteria, including XDR-TB.
Further, SQ109 synergizes with other TB drugs in experimental animal models of TB, and could result in new TB drug combinations that have much greater activity than the current standard of care.
"We are pleased that the FDA and the EMEA awarded Sequella orphan drug and orphan medicinal product status for the use of SQ109 (N-adamantany1-N'-Geranyl-ehtylendiamine) for the treatment of tuberculosis (TB), a chronically debilitating and life-threatening infectious pulmonary and extrapulmonary disease," said Dr. Carol Nacy. "With the resurgence of TB in a new drug-resistant form it is clear there is a need to readdress the treatment of this infectious and deadly disease. To accelerate our clinical programs, we will continue to work closely with the FDA's Office of Orphan Products Development and the EMEA's Committee for Orphan Medicinal Products as we move through the clinical and regulatory process."
The Orphan Drug Act (Public Law 97-414, enacted in 1983 and amended) provides for economic incentives to encourage pharmaceutical companies to develop drugs for rare diseases defined as those affecting fewer than 200,000 people in the United States.
Orphan drug designation entitles Sequella, Inc. to seven years of market exclusivity for SQ109 for the treatment of patients with tuberculosis. Additional incentives for orphan drug development include tax credits related to certain development expenses, an exemption from the FDA user fee, and FDA assistance in clinical trial design. Importantly, the Orphan Drug Act facilitates a close working relationship between regulatory agencies and companies aimed at accelerating the drug development and approval processes for treatment of rare diseases.