Roche's PIASKY Approval to Challenge AstraZeneca in Paroxysmal Nocturnal Hemoglobinuria Treatment

Following significant achievements in Asia, Roche's PNH treatment drug PIASKY (crovalimab) has received its first FDA approval. This milestone has intensified the competition between Roche and the current market leader, AstraZeneca, with other pharmaceutical companies also gearing up to introduce their lead assets.

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare condition characterized by three main features: intravascular hemolysis, thromboembolic events, and cytopenia. Due to its unpredictable nature, patients exhibit a wide range of symptoms, complicating classification based on typical presentations.

In 2023, approximately 12,000 cases of PNH were diagnosed across the seven major markets (7MM), with the numbers expected to rise to around 13,000 by 2034. The United States recorded the highest number of diagnosed cases. Before 2007, PNH was particularly devastating due to the lack of effective treatments for hemolysis and thrombosis, the leading causes of death in patients. The introduction of eculizumab, an anti-C5 agent, marked a revolutionary shift, significantly reducing hemolysis, transfusion dependency, and thrombosis rates.

Treatment strategies for PNH now focus on complement inhibition therapy. Drugs like SOLIRIS, ULTOMIRIS, and EMPAVELI, all FDA-approved, are considered the gold standard. SOLIRIS, developed by Alexion Pharmaceuticals, was the pioneering therapy and was succeeded by ULTOMIRIS. Both drugs offer essential C5 inhibitors as therapeutic options.

Recent approvals of factor B and D inhibitors such as FABHALTA (iptacopan) in the US and VOYDEYA (danicopan) in Japan aim to provide better treatment with fewer side effects. The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) recommended crovalimab for treating PNH in adults and adolescents aged 12 and above in June 2024, based on results from the global Phase III COMMODORE 2 clinical trial. 

In September 2023, the FDA accepted a biologics license application for crovalimab and approved it in June 2024 for treating PNH in adults and adolescents aged 13 and older. Genentech, a Roche subsidiary, has made the drug available in the US. In Japan, Chugai Pharmaceutical submitted a new drug application for PiaSky to the Japan Ministry of Health, Labour, and Welfare (MHLW) in June 2023, which was approved in March 2024 and launched in May 2024 for patients aged 12 years and older. 

China's National Medical Products Administration (NMPA) also approved crovalimab for treating PNH in adults and adolescents aged 12 and older who have not previously received complement inhibitor treatment in February 2024, after a priority review that started in August 2022.

PiaSky is currently under review by other regulatory authorities, including those in Europe and Taiwan. The PNH market is highly competitive, with numerous companies working in the field. Emerging therapies such as Pozelimab (REGN3918) combined with Cemdisiran, OMS906, and NM8074 (ruxoprubart) highlight the dynamic evolution of treatment strategies.

Pozelimab, a novel human monoclonal antibody, aims to inhibit complement factor C5, preventing the destruction of red blood cells responsible for PNH symptoms. This IgG4 antibody binds effectively to both wild-type and variant forms of human C5, blocking its function. It is also being studied in combination with Alnylam's cemdisiran, a siRNA C5 inhibitor, to treat various complement-mediated disorders, including PNH and myasthenia gravis.

OMS-906, another emerging therapy, is being developed to treat PNH, complement 3 glomerulopathy (C3G), idiopathic immune complex-mediated glomerulonephritis (ICGN), and arthritis. It can be administered subcutaneously or intravenously, targeting mannan-binding lectin serine protease 3 (MASP-3) to achieve its therapeutic effects.

These emerging therapies are expected to transform the PNH market landscape in the coming years, offering new standards of care and unlocking opportunities for medical innovation and economic growth. In 2023, the total PNH market size in the 7MM was valued at approximately USD 1.4 billion, with projections suggesting it could reach around USD 2.5 billion by 2034. This growth is driven by advances in disease mechanisms, new diagnostics, and therapeutic developments, alongside the increasing prevalence of PNH and the introduction of new therapies.

How to obtain the latest research advancements in the field of biopharmaceuticals?

In the Synapse database, you can keep abreast of the latest research and development advances in drugs, targets, indications, organizations, etc., anywhere and anytime, on a daily or weekly basis. Click on the image below to embark on a brand new journey of drug discovery!