Cycle Pharmaceuticals: The Company Behind the First FDA-Approved Drug for Alkaptonuria

Alkaptonuria (AKU) is an ultra-rare condition that’s characterized by skin discolouration, arthritis and increased risks of heart failure and stroke. While those suffering from this disease can go the entirety of their lives without treatment, doing so can be detrimental to their quality of life.  

For patients with this condition, an exciting announcement took place in June of this year. The FDA has approved the first ever alkaptonuria drug. Having just been released in July 2025, this product launch marks the first time patients will have something other than lifestyle modifications to turn to.  

Nitisinone, branded Harliku, has been developed by biotechnology company Cycle Pharmaceuticals. With a focus on rare genetic diseases, this win reinforces their mission of committing to and fully supporting the AKU community as well as empowering rare disease patients. To quote them, “every single patient matters”.  

The complications in treating AKU 

AKU is a metabolic disorder that causes patients to have a buildup of homogentisic acid (HGA). Over time, this can lead to osteoarthritis, skin discolouration, heart complications and kidney problems – all of which can affect emotional well-being, physical abilities and overall quality of life.  

Unfortunately, treating AKU is often an arduous task due to a whole range of clinical and practical complications. Aside from its sheer rarity, one of the main difficulties is simply due to its slow rate of progression.  AKU symptoms often go unnoticed – they are particularly subtle during early life and childhood – which means the majority of cases aren’t diagnosed until adulthood. By then, irreversible joint and tissue damage will already have begun.  

For decades, there have been no specific treatment options for AKU. Patients have been recommended to manage their symptoms through diet changes, avoiding certain physical activities and taking pain medication. But now, the approval of Harliku acts as the big first step in providing AKU patients a drug that targets the disease itself.   

The approval of Harliku  

Harliku was approved following a randomized, no-treatment control study of 40 patients with AKU. According to the Medical Professionals Reference, findings showed that “after one year of treatment with nitisinone, the average percent reduction in urinary HGA from baseline was 88%. Conversely, the untreated control group had an average increase of 107%”. Trial results also found that patients treated with Harliku experienced improvements in pain, physical functionality and energy levels after three years of treatment.  

The small molecule drug works by “lowering the amount of HGA in the urine of people with AKU by inhibiting an early step in the body’s process of breaking down protein”. By slowing down the rate of disease progression, it offers patients an improved quality life that hasn’t previously been available.  

It’s wonderful to see rare diseases like alkaptonuria receiving the attention they need, where patients finally have a treatment option to turn to.  

Cycle Pharma and their impact on rare diseases  

Harliku isn’t the only successful drug developed by Cycle Pharma. In fact, they have developed an impressive list of products for a whole range of rare diseases. This includes treatment for phenylketonuria (PKU), tyrosinemia type 1 (HT-1), hereditary angioedema (HAE), primary periodic paralysis (PPP) and cystinuria. They have also developed their own version of a common multiple sclerosis treatment that comes in the form of a unique orally disintegrating tablet.   

The biotech has also created Cycle Vita, a patient support programme for people living in the United States. For those who are prescribed any of their medications, Cycle Vita offers personalized emotional, financial and educational support.  

Their health tech platform gives individuals the chance to take better control of their own treatment and understand their condition, which ultimately sets them up to take better care of themselves and improve their quality of life in the long run. 

Final note 

The work done by Cycle Pharma is making a tremendous difference for those affected by rare diseases; a collective of people so frequently lacking proper treatment options.   

However, it also acts as a reminder that in-depth research into rare diseases must continue so that reliable drugs can be brought to hundreds of other patients who are awaiting sought after treatments.  

Meet takes pride in helping life sciences organizations build the teams that contribute to life-changing medicines and medical devices. From clinical stage biotechs to well-established pharma companies, we help to place the people that create trial wins and exciting advancements that improve global health outcomes.   

If you’re looking to expand an important team or take the next pivotal step in your career journey, please reach out to us today.