Amgen was aiming to launch its cholesterol-fighter Repatha quickly in India. But a panel of CV and renal specialists blocked the PCSK9 drug instead.
The U.S. biotech was aiming to import and market Repatha with the help of a waiver exempting the drug from pre-launch testing in Indian patients. But the Central Drugs Standard Control Organization (CDSCO) panel denied that request, delaying approval and entry into the fast-growing Indian pharma market.
India's clinical trial waiver scheme was developed by the CDSCO as a means of speeding introduction of certain drugs already licensed in other "well-regulated" markets such as the U.S. and EU.
Under the scheme, drugs for rare diseases and others with few treatment options can be approved without a Phase III trial in India, provided drugmakers carry out post-marketing studies to monitor their safety and efficacy. Before granting a waiver, a CDSCO Subject Expert Committee (SEC) assesses the application and surveillance plan.
In Repatha's case, the cardiovascular and renal SEC found that the drug appeared to be safe and fulfilled the criteria of orphan drug status as a treatment for homozygous familial hypercholesterolemia (HoFH). However, the panel also said the Amgen drug has "a very high potential for misuse in conditions other than the orphan condition."
Repatha is approved for broader use in the U.S. and EU. There, it's also targeted at patients unable to control their cholesterol despite aggressive statin therapy or who cannot take statins.
Last year, Amgen enlisted India-based Dr. Reddy's Laboratories to speed up registration and marketing for Repatha and two other growth products, the multiple myeloma drug Kyprolis and immunotherapy Blincyto. Now, the SEC verdict has thrown the timeline for Repatha into disarray.
Amgen was tight-lipped on the delay, limiting itself to saying that it would "continue to engage with the Indian regulatory authorities to make Repatha … available to patients who need this therapy in India, as soon as possible."
Repatha is one of the products expected to drive growth at Amgen in the coming years but--along with rival PCSK9 inhibitor Praluent from Sanofi/Regeneron--has been struggling to make headway in western markets.
A high list price of $14,000-plus a year and prescribing barriers set up by healthcare payers to limit uptake have taken their toll, and despite multibillion-dollar sales projections for both Repatha and Praluent, initial take-up has been disappointing. Amgen reported Repatha sales of $43 million in the first 6 months of the year, while Praluent brought in around $40 million in the same period.
Both Amgen and its rivals are hoping data from cardiovascular outcomes trials will inject some momentum into the drugs, and these studies may also help them access emerging markets. Sanofi/Regeneron's ODYSSEY trial and Amgen's FOURIER study have recruited Indian patients so potentially could be used to support approvals in India.
- read the CDSCO committee report (PDF)
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