The partnership, announced today (January 18) will make NITYR (nitisinone) tablets available for eligible patients in the UK with Hereditary Tyrosinemia Type 1 (HT-1) and Alkaptonuria (AKU).
A Free Goods Programme is a compliant way for medicines that are not approved in their country of intended use to be made available for patients who either have no alternative treatment option or have exhausted all other treatment options available in their country of residence.
Stuart Bell, senior vice president, Inceptua early access said: “Inceptua is proud to partner with Cycle in this Free Goods Programme to allow access to NITYR to a broader number of countries and patients. Cycle’s ethos around medicine provision aligns very closely to those of Inceptua’s, and we look forward to supporting patients with no other treatment options for these devastating diseases.”
Rare genetic condition
Ht-1 is a very rare genetic condition. It prevents the body from breaking down a substance called tyrosine found in food. This leads to the build-up of toxic levels of substances in the blood. If left untreated, TYR1 can damage the liver, kidneys, and the nervous system.
The NHS describes AKU, or black urine disease, as a very rare inherited disorder that prevents the body fully breaking down two protein building blocks (amino acids) called tyrosine and phenylalanine. It results in a build-up of a chemical called homogentisic acid in the body. This can turn urine and parts of the body a dark color and lead to a range of health problems over time.
Cycle has an existing Free Goods Program with patients with HT-1 in India, Bangladesh, Pakistan, and Sudan. Inceptua will support Cycle through the expansion of the Free Goods Programme to include further countries where NITYR is not commercially available, or where the local healthcare system is unable to afford this medication.
Countries in scope of the NITYR Free Goods Programme include Argentina, Bangladesh, Brazil, Chile, Colombia, Dominican Republic, Jordan, Mexico, Nigeria, Pakistan, Peru, Tunisia, and Turkey. Cycle says it is committed to supporting patients on the Free Goods Programme for the duration of their lifetime.
Proud and humbled
James Harrison, CEO of Cycle Pharmaceuticals said: “Right from the beginning of Cycle’s story in 2012, we made a commitment to provide a Free Goods Programme for rare disease patients that cannot otherwise access our medicines. The whole Cycle team is extremely proud and humbled to be able to expand the NITYR Free Goods Programme. It really matters.”
In its research, the companies found that HT-1 affects about 1 in 100,000 individuals worldwide, whilst AKU affects 1 in 250,000 to 1 million people worldwide.
NITYR (nitisinone) tablets are a competitive inhibitor of 4-hydroxyphenyl-pyruvate dioxygenase indicated for the treatment of adult and pediatric patients with HT-1 in combination with dietary restriction of tyrosine and phenylalanine. NITYR has been approved for use by the FDA, Health Canada, EMA, MHRA and TGA.