U.K. MHRA Awards Tern Therapeutics Innovation Passport for TTX-381 to Accelerate Gene Therapy for the Treatment of the Ocular Manifestations of CLN2 Disease
MHRA granted Tern Therapeutics’ TTX-381 an Innovation Passport under the accelerated Innovative Licensing and Access Pathway (ILAP)
ILAP is designed to optimize the development and expedite the evaluation of innovative medicines for patients with high unmet need
TTX-381 has also previously been granted Regenerative Medicine Advance Therapy (RMAT) and Fast Track Designations by the U.S. Food and Drug Administration (FDA)
Tern Therapeutics, LLC (“Tern”), a biotechnology company developing transformative one-time gene therapies for rare diseases, today announced that TTX-381 has been awarded the Innovation Passport, the United Kingdom Medicines and Healthcare products Regulatory Agency (MHRA) innovative medicine designation. TTX-381 is a novel one-time investigational gene therapy product being developed for the treatment of the ocular manifestations of CLN2 disease, a form of Batten disease.
“The CLN2 community urgently needs treatment for the loss of vision caused by CLN2 disease,” said Alex Bailey, Ph.D., Chief Executive Officer of Tern. “Following the recent RMAT and Fast Track designations of TTX-381 by the FDA, the Innovation Passport designation from the MHRA underscores our commitment to delivering TTX-381 to patients around the world as quickly as possible. We look forward to continued collaborations with U.K. health authorities to help us achieve this goal.”
“We are pleased with the rapid progress we have made enrolling the TTX-381 clinical trial at Great Ormond Street Hospital (GOSH) and the promising results emerging from the study,” said Robert Henderson, MD. “ILAP designation highlights the key role of the MHRA and our team at GOSH in developing this product and accelerating its availability to people around the world living with CLN2.”
The Innovation Passport is the entry point to the ILAP, which aims to accelerate time to market for transformative new medicines and facilitate patient access to medicines in the U.K. This designation provides Innovation Passport holders with the opportunity to work more closely with the MHRA and its partner agencies, including the National Institute for Health and Care Excellence (NICE), and grants access to numerous programs designed to support innovative products, such as Joint Scientific Advice with MHRA, NICE, and other partners as needed, as well as priority scheduling for key regulatory and Health Technology Assessment support services. Other benefits of ILAP include the potential for a 150-day accelerated Marketing Authorization Application assessment, a rolling review, and a continuous risk-benefit assessment.
MHRA awarded TTX-381 the Innovation Passport based on three criteria: that the condition addressed is seriously debilitating with a significant unmet clinical need; it is innovative; and that it has the potential to substantially improve patient health outcomes.
