US FDA Grants Once Daily Dosing Option for NITYR (nitisinone) Tablets for HT-1 Patients

September 14th 2020

BOSTON, Mass. September 14th, 2020 – Cycle Pharmaceuticals is pleased to announce that the U.S. Food and Drug Administration (FDA) has approved the once-daily dosing of NITYR®.

This dosing regimen is now an option for patients 5 years of age and older who have undetectable serum and urine succinylacetone concentrations after a minimum of 4 weeks on a stable dosage of nitisinone.

NITYR® is indicated for the treatment of patients with hereditary tyrosinemia type 1 (HT-1) in combination with dietary restriction of tyrosine and phenylalanine.

HT-1 is an ultra-rare genetic disease that can cause hepatic, renal and peripheral nerve damage. In most cases, if left untreated, the disease is fatal. It is estimated that there are approximately 200 patients in the U.S.

“The inclusion of the once daily dosing option in the NITYR Prescribing Information provides NITYR’s patients and their prescribers with greater flexibility in dosing and titration. This new option may be more convenient to patients and their caregivers in their daily management”  Peter Myrenfors, Chief Medical Officer, Cycle.

NITYR® is a small and tasteless tablet (baby aspirin sized) for all ages launched by Cycle in the US in 2017 and now approved in many countries around the world. NITYR® can be tried for 30 days by U.S. patients through the Free NITYR® Program1, at the end of which they can freely decide to stay on NITYR®, or to go back to their previous treatment.

“We are proud to offer another great improvement to the NITYR experience for our patients with HT-1. This result is part of Cycle’s continuous investment and efforts in widening options for patients and caregivers and reinforces our commitment to the HT-1 community.” said Zac Bohlen, Head of Operations in the US, Cycle.

For full Prescribing Information and important safety information, please visit www.nityr.us/PI.

References: 1. Free NITYR Program: Patients can try NITYR for 30 days, one time, for free, as long as their doctor prescribes it. Subject to terms and conditions, eligibility criteria, and other federal and state law. 2. Wellbeing Program (Low-Protein Food Program and Online Fitness Program): Subject to terms and conditions and eligibility criteria. This program is for eligible commercially insured patients only. Not available for government-insured patients and subject to other federal and state law.

Terms and Conditions and eligibility criteria for all current programs are available at www.nityr.us/offerstcs.

 

About Cycle Pharmaceuticals

Cycle Pharmaceuticals was founded in 2012 with the sole aim of delivering best-in-class drug treatments and services to the under-served rare disease patient community. We focus on rare metabolic and neurological genetic conditions.  Cycle is headquartered in Cambridge, UK and has offices in Boston, Mass. (USA).  For more information, please visit www.cyclepharma.com and follow us on Twitter, LinkedIn, Facebook and Instagram.