NEW DELHI: Patients with difficult-to-control high cholesterol could soon have an oral alternative to powerful cholesterol-lowering injections after the US Food and Drug Administration (FDA) approved the world’s first oral PCSK9 inhibitor, a class of medicines that dramatically lowers “bad” cholesterol.The once-daily tablet, Lipfendra (enlicitide), has been approved for adults with high cholesterol, including those with an inherited condition called heterozygous familial hypercholesterolaemia (HeFH). The drug is to be used along with diet, exercise and existing cholesterol-lowering treatment. Until now, PCSK9 inhibitors have only been available as injections administered every two or four weeks. However, the drug is not expected to be available in India immediately.“Even if a medicine is approved abroad, it cannot be marketed in India automatically. Besides patent-related restrictions, it must obtain approval from the CDSCO after following the prescribed regulatory process, which may include clinical trials as required under Indian regulations. In exceptional cases, patients may seek permission through a separate CDSCO mechanism to import limited quantities for personal treatment. However, medicines cannot simply be brought into the country by friends or relatives,” said Dr Subhash Mandal, national general secretary, Indian Pharmaceutical Association (IPA).The approval is significant because many high-risk patients continue to have elevated low-density lipoprotein (LDL) cholesterol despite treatment with standard oral medicines. Indian cardiology guidelines recommend aggressive LDL reduction, with targets below 70 mg/dL for high-risk individuals and below 55 mg/dL for those at very high cardiovascular risk, but many patients fail to achieve these goals despite multiple therapies.With cardiovascular disease remaining India’s leading killer, cardiologists say better LDL cholesterol control is key to preventing heart attacks and strokes. Clinical trials involving 3,207 adults found the once-daily oral drug reduced LDL cholesterol by 56-59% over 24 weeks in patients already on maximally tolerated statins, delivering reductions comparable to injectable PCSK9 inhibitors and offering another option for those whose cholesterol remains uncontrolled despite existing therapy.However, cardiologists cautioned that the drug is unlikely to replace statins or become a first-line treatment.“PCSK9 inhibitors have so far been available only as injections every two or four weeks. The new oral therapy lowers LDL, or ‘bad’ cholesterol, by about 50-60%, making it a promising option for patients who prefer to avoid injections,” said Dr Ambuj Roy, Professor of Cardiology at AIIMS Delhi.He said the drug is likely to be used as a fourth-line therapy, after statins, ezetimibe and bempedoic acid, for patients whose LDL cholesterol remains uncontrolled or who cannot tolerate statins. “While it lowers LDL substantially, we still lack long-term evidence that it reduces heart attacks, bypass surgery, stenting or deaths. It is also expected to be expensive,” he said.High LDL cholesterol is a major risk factor for heart disease and stroke. It causes fatty plaques to build up inside arteries, increasing the risk of heart attacks and strokes.
