Cycle Pharmaceuticals Receives Approval for Updated Infant Administration for NITYR (nitisinone) Tablets for HT-1 in U.S.

NOVEMBER 12, 2018

BOSTON, Mass. November 12, 2018 – Cycle Pharmaceuticals (Cycle) is pleased to announce that the U.S. Food and Drug Administration (FDA) has approved a new version of the NITYR™ Prescribing Information including an improvement to the administration procedure for infants using an oral syringe.

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.

NITYR™ is a small and tasteless tablet (baby aspirin size) for all ages. For patients, including pediatric patients who have difficulty swallowing, the tablets can also be disintegrated in water and administered using an oral syringe.

The Prescribing Information has been updated to allow caregivers to disintegrate a NITYR™ tablet in an oral syringe up to 24 hours before administration.

“We have worked with caregivers, physicians and the FDA to simplify the way that NITYR™ can be administered to children using an oral syringe, until they are able to swallow the small tablet as a whole. By increasing the time to administer the preparation of the suspension from 2 hours to 24 hours, we are giving families a full day to decide when is the right time for them to administer the medication to their children, and in this timeframe, they are free to carry on with their daily commitments.  We think that giving the caregivers this improvement is further contributing to their quality of life. Moreover, disintegrating the tablets prescribed in an oral syringe, will allow an accurate dosing, avoiding possible risks of under or over dosing” said Peter Myrenfors, Chief Medical Officer, Cycle.

 Cycle’s nitisinone tablets were recently approved by the European Medicines Agency (EMA) in Europe and it is currently prescribed in key countries and regions around the world such as U.S. and Canada, Middle East, Australia, Latin America, South East Asia and South Africa.

“We are very pleased at Cycle for this approval, as it represents another step forward to improve HT-1 patients’ quality of life. To further improve NITYR™, Cycle will continue investing and widening options for our patients in the future. We are grateful to the U.S. healthcare professionals and the HT-1 community for their remarkable response and willingness to welcome innovation since we launched last year” said Antonio Benedetti, CEO, Cycle

In clinical trials, NITYR™ tablets have an established safety profile with warnings and precautions as follows:

  1. Elevated Plasma Tyrosine Levels, Ocular Symptoms, Developmental Delay and Hyperkeratotic Plaques: Inadequate restriction of tyrosine and phenylalanine intake can lead to elevations in plasma tyrosine, which at levels above 500 micromol/L can result in symptoms, intellectual disability and developmental delay or painful hyperkeratotic plaques on the soles and palms; do not adjust the dosage of NITYR in order to lower the plasma tyrosine concentration. Obtain slit-lamp examination prior to treatment and re-examination if symptoms develop. Assess plasma tyrosine levels in patients with an abrupt change in neurologic status.
  2. Leukopenia and Severe Thrombocytopenia: Monitor platelet and white blood cell counts.

There are no contraindications for NITYR™ tablets, however, patients should consult their Doctor prior to taking this medication. For full prescribing information and important safety information on NITYR™, please visit www.nityr.us/PI.

About Cycle Pharmaceuticals, the commitment to HT-1 and NITYR

Cycle Pharmaceuticals is committed to changing the way that HT-1 is treated and managed around the world. Cycle Pharmaceuticals has worked for years with the FDA, physicians and patient groups to reduce the impact that HT-1 treatment has on patients’ lives. NITYR™ is the first and only nitisinone tablet in the world that does not require refrigeration (removing the need for cold chain transportation and storage) and can be taken with or without food; which may represent a significant improvement on previous treatments for patients and caregivers. NTIYR™ has now a shelf life of 3 years. NITYR™ can be tried for free and has a unique Diet and Low Protein Food Program.