This small biotech notched a big win for rare disease patients with a canny market access strategy

Immunoglobin A nephropathy (IgAN), also known as Berger’s disease, is a rare autoimmune disease of the kidneys that can, over 10 to 20 years, progress to end-stage renal disease. Clarivate forecasts the IgAN therapy market will grow at a CAGR of 26% to $4.2 billion in 2033, largely fueled by the emergence of novel therapies along with greater disease awareness and treatment rates, with patient populations projected to exceed 180,000 in the U.S. and 200,000 in the EU5 (Clarivate Disease Landscape & Forecast, September 2024).

Until recently, treatment was strictly symptomatic, and there remains high unmet need for therapies that address kidney function decline, particularly those that more effectively lower UPCR and slow the decline in eGFR. The entry of TARPEYO® (budesonide) to the U.S. market in 2021 was a game changer. The drug, a delayed-release formulation of the corticosteroid budesonide from Calliditas Therapeutics in partnership with STADA Arzneimittel AG and Everest Medicines, is currently the only FDA-approved treatment for IgAN shown to significantly reduce the loss of kidney function – phase 3 trials showed 50% less damage to the kidneys compared to placebo over two years.

For a smaller company like Calliditas, commercializing a new drug and ensuring patients can access it presents a daunting challenge, leading many biotechs to sell or license their precious innovations (and with them, the lion’s share of the spoils, which could fund future innovations) to large pharma while still in early stages of clinical trials. However, with TARPEYO, Calliditas was able to utilize the FDA’s Orphan Drug Designation pathway to win an Accelerated Approval.

From a very limited initial indication – to reduce proteinuria (elevated protein in the urine) in adults with primary IgAN at risk of rapid progression – the company was able to win an expanded label based on follow-up clinical trial data, now covering a reduction in loss of kidney function for that patient population. Research on the use of the drug for other indications, including autoimmune hepatitis (AIH) and primary biliary cholangitis, is underway. However, given its high cost and new competitors emerging, TARPEYO’s dominance of the IgAN treatment marketplace will be challenged in the years to come.

Clarivate analysts charted TARPEYO’s path to market as part of a new series of reports looking at the market access strategies taken by some of our 2024 Drugs to Watch. Read our full report on the regulatory journey of TARPEYO/Kinpeygo, including analysis of the broader competitive and coverage landscape around IgAN, here. We have also taken a look at the market access strategies of AREXVY and ABRYSVO™ and Eylea® HD.

To learn more about how Clarivate helps innovative companies navigate a turbulent global market access landscape, please visit us here.

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