Release Date: 08-Feb-2023
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired blood disorder wherein blood cells die prematurely and their production in impaired. The disorder affects both red and white blood cells as well as platelets, and results in symptoms such as fatigue, paleness and tachycardia in children. Being a rare disorder having a small patient base, paroxysmal nocturnal hemoglobinuria had not been a major focus for pharma giants until about two decades ago when Alexion Pharmaceuticals launched the first drug Soliris targeting the C5 protein for the treatment of this rare blood disorder. The sales of this drug were high and as a result more C5 inhibitors entered the pipeline and are showing promising results.
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The most recent results for an investigational C5 inhibiting drug to be revealed were for Crovalimab, under investigation by Roche for paroxysmal nocturnal hemoglobinuria. Results from a pivotal phase III trial titled COMMODORE 2 which was initiated in 2021 for patients with the blood disorder who have not been treated previously with complement inhibitors showed positive results. Data from another separate phase III COMMODORE 1 study where patients with paroxysmal nocturnal hemoglobinuria using currently approved complement inhibitors were switched to Crovalimab, which concluded with data supporting the favorable benefit-risk profile of Crovalimab as was reported from the first study.
Soliris, or Eculizumab, is currently considered the standard of treatment for paroxysmal nocturnal hemoglobinuria but results from Roche’s COMMODORE 1 and COMMODORE 2 studies demonstrated that Crovalimab was non-inferior to Eculizumab. Crovalimab was developed keeping in mind the comfort of patients; as a result, the drug has been developed as a subcutaneous injection which is administered every four weeks unlike Eculizumab which is given every two weeks as an intravenous injection. Roche is optimistic about Crovalimab and plans to submit data from both COMMODORE 1 and COMMODORE 2 studies to drug regulatory authorities around the world to get approval for the condition and will also present the data at an upcoming medical meeting.
Approval of Crovalimab for paroxysmal nocturnal hemoglobinuria will help patients by giving them another option for treatment that too at a less frequent dosage interval and submission of data to regulatory authorities has brought the drug a step closer to getting an approval and patients suffering from disorders involving C5 activation. Apart from paroxysmal nocturnal hemoglobinuria, Crovalimab is also being evaluated in atypical hemolytic uremic syndrome and sickle cell disease, also rare diseases where blockade of the C5 protein has proven to be beneficial. Clinical trials for both these indications are in their second and third phases respectively. While sickle cell disease has no drug approved for treatment, bone marrow or stem cell transplants are the only viable cure for the diseases where Alexion’s Soliris (Eculizumab) is also approved for atypical hemolytic uremic syndrome treatment. Results from Roche’s undergoing trials will help determine the better of these two treatment candidates.
Rare diseases are diseases with a very low prevalence as compared to other prevalent diseases in the general population. Paroxysmal nocturnal hemoglobinuria is one such disease which has an estimated prevalence of a mere 0.5-1.5 per million people in the global population. The disease is believed to affect people of both genders equally; however some reports have shown the disorder an inclination towards females. In the presence of only a limited number of affordable treatment options, Crovalimab has come up as a feasible treatment option, having an edge over Eculizumab with less frequent dosing, which may translate to less spending on the overall treatment. However, this may be only determined when the price for the treatment is revealed by Roche.
The market for rare diseases is huge considering out of the 7000 known rare diseases, approximately only 5 percent of these have a treatment. Patients are either receive treatment to manage their symptoms or are often treatment with off-label drugs which may not be as effective. Steps taken by companies such as Alexion, Roche Apellis and Novartis have brought these diseases into focus and the overall pipeline for rare diseases has been slowly expanding, giving hope to patients suffering from these diseases because of more modern targeted approaches being used for their treatment. The pipeline is anticipated to expand further in the coming times as the market size continues to grow, coupled with the steps different central governments and drug regulatory authorities have been taking to encourage drug development for different rare diseases.