Novartis nabs first is the FDA nod for rare kidney disease, but Apellis Pharma follows
Now, an ultra-rare kidney disease that can be life-threatening has been adopted for FDA-approved treatment. The affirmative regulatory decision is the third item of Novartis’ Fabhalta, establishing the potential potential of the drug in the product. However, competition comes from competitors’ drugs with similar approaches.
Fabhalta's March 20 approval covers the treatment of adult C3 glomerulopathy (C3G), which affects the glomerulus, tiny blood vessels in the kidneys that are responsible for filtering waste from the blood. C3G can develop renal failure. So far, standard treatments include supportive care, extensive immunosuppression and symptom management, as there are no specialized treatments for the disease.
C3G originates from the pathways of complement system (part of the immune system) that are too active. Overactivation of this pathway leads to the deposition of complement protein C3 in the glomerulus, triggering inflammation and kidney damage. Fabhalta is an oral small molecule designed to bind and block alternative complement proteins. Factor B. Pills twice a day were first approved in 2023 as a treatment for paroxysmal nocturnal hemoglobinuria (PNH). Last year, the drug expanded its approval to include rare autoimmune kidney disease IGA nephropathy.
The Fabhalta submission submitted by Novartis in C3G is a placebo-controlled 3-phase test that evaluates the study medication along with supportive care. Compared to placebo, twice daily medications resulted in a 35% reduction in proteinuria, and urine protein levels were indicative of kidney disease. This reduction was observed 14 days after initiation of treatment. The therapeutic effect lasted at 12 months.
The most common adverse reactions reported in key studies are nasopharyngitis and viral infection. The drug’s label issued a black box warning to patients that they might develop severe infections due to encapsulation of bacteria, a warning that the drug was initially approved in PNH. Fabhalta can only be obtained through a restrictive program that manages these risks. Patients must be vaccinated with encapsulated bacteria at least two weeks before the first Fabhalta dose, and clinicians should monitor the patient for signs of severe infection.
Novartis expects Fabhalta to reach a peak sales of $3 billion in several signs. It still has a way. According to the company's annual report, the drug's revenue in 2024 was $129 million. To expand the use of drugs and maximize their revenue potential, Novartis is conducting clinical studies on other rare renal diseases, such as immune complex membranous proliferative glomerulonephritis (IC-MPGN).
Fabhalta, used in its approved PNH, has competed with Empaveli, which injects Apellis Pharmaceuticals twice a week, blocking the complement system protein C3. Both drugs may also become competitors to C3G and IC-MPGN. Apellis has filed an application with the FDA to seek to expand approval for its drug to both signs. The document is based on the results of a Phase 3 study that showed that the drug resulted in a 68% reduction in proteinuria measured six months later. The drug is being developed under a partnership with SOBI; Swedish company has exclusive rights to commercialize systematic empaveli outside the United States
Fabhalta's new approval of empaveli's C3G Bodes is good for Empaveli, and his data suggests that it's the best-in-class for C3G and IC-MPGN, William Blair analyst Lachlan Hanbury-Brown said in a note sent to investors on Friday. Clinicians told the company that their choice of treatments for these rare renal diseases will be driven by efficacy rather than potential patient preference for oral therapy. The efficacy measures are obviously beneficial to Empaveli.
“To date, there are approximately 5,000 patients in the United States, with no approved therapies (and still no treatments for adolescents with IC-MPGN or any disease), and a distinction between product profiles, which we consider to be a significant market opportunity for Apellis.”
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