How new oral pill Lipfendra can be a game changer for bad cholesterol 

Whether it’s cost, cold-chain storage, needle aversion or the need for repeated administration, “a pill removes most of that friction”, says Dr Vishal Khullar, director, cardiovascular and thoracic surgery, heart and lung transplant, Fortis Hospital Mulund, Mumbai. “It moves this class from tertiary cardiology centres into the hands of the general physician and family doctor, which is where the majority of dyslipidaemia is actually managed,” he adds.

Dyslipidaemia (or hyperlipidaemia) is a condition for which the burden is rising globally, including in India. So far, only an expensive injectable drug was available, but with the recent approval, the condition can now be managed with a prescribed pill, at least in the US. Doctors say this could make a marked difference in the treatment and management of this bad cholesterol burden, should the drug be approved for distribution in India as well.

Potential impact

The significance here is not that we have discovered a new way to lower cholesterol, say experts. “PCSK9 inhibition has been available for close to a decade, and we already know it works: evolocumab and alirocumab (sold as injectable medicines to block the protein and enzyme in order to lower LDL cholesterol) have proven cardiovascular benefit in large outcome trials,” says Dr Khullar. “The significance is that this mechanism has finally been packaged into a tablet.”

Dr Amit Kumar Malik, director, interventional cardiology and electrophysiology at Medanta Noida, explains further, “An oral option is likely to improve convenience, treatment acceptance, and long-term adherence, especially among patients who are reluctant to take injections.”

Clinical studies have shown that it can reduce LDL cholesterol by nearly 60%, comparable to injectable PCSK9 inhibitors. If patients remain on therapy consistently, it has the potential to reduce the long-term burden of heart attacks and strokes.

“An oral medicine has several practical advantages. Unlike injections, it does not require refrigeration or specialised administration, making it easier to prescribe, distribute, and use in routine clinical practice. Whether it becomes truly affordable in India will depend on pricing and market competition. If these factors are favourable, wider adoption among eligible high-risk patients is certainly possible,” Dr Malik adds.

Injectables have one underrated advantage, though, notes Dr Khullar. Someone administers them, so adherence is essentially guaranteed for that interval. “A six-monthly injection means the patient is protected for six months whether or not they remember. A daily pill goes onto a tablet pile that many of my patients already find overwhelming, and we know how poorly daily statin adherence holds up beyond the first year,” he adds.

Hidden danger

Dr TS Kler, chairman of BLK-Max Heart and Vascular Institute, sheds light on the burden of LDL cholesterol: “As far as South Asia is concerned, including India, the problem is that LDL is high, while HDL (high-density lipoprotein), which is the protective cholesterol, is low. One of the most important factors causing heart disease is increased LDL, or bad cholesterol.” He says that in situations where statins are not effective due to the severity of the condition “or are causing significant side effects, this medicine will be of great benefit”.

“In India, many people do not know their cholesterol or LDL levels until they suffer a heart attack. Annual health check-ups should begin at a young age so that we know a person’s lipid profile early and can take appropriate action,” he adds.

According to Dr Khullar, the numbers in India are sobering. “A nationwide ICMR-INDIAB study found dyslipidaemia in roughly 81% of adults screened, with low HDL cholesterol in about two-thirds and elevated LDL in one in five.”

“The burden is severe and, more importantly, it is largely invisible. High cholesterol shows no symptoms until it produces a catastrophe,” he warns. “Cardiovascular disease is already the leading cause of death in India, and Indians develop it younger and with more aggressive disease patterns than most other populations.”

Speaking from his own experience with patients, he says, “I see the end result of this on my operating table. I am regularly performing bypass surgery on men in their late 30s and 40s – patients whose fathers had their first heart attack at 50. Familial hypercholesterolaemia, which Lipfendra is specifically approved for, is estimated to affect around one in 250 people worldwide, which would put the Indian figure well over half a million – and the overwhelming majority of them have never been diagnosed,” adds Dr Khullar.

Indian context

What kind of potential is this drug going to have in the Indian market? “The introduction of any new medicine in India depends on regulatory approvals, commercial decisions and clinical considerations. India has a strong pharmaceutical sector, and over time newer therapies often become available through different pathways, but it is too early to comment on any specific product or manufacturer,” says Dr SK Wangnoo, senior consultant, endocrinology, Indraprastha Apollo Hospital, Delhi.

On how the pill is different from the injection, Dr Wangnoo says that expanding the range of treatment options supports a more personalised approach to cholesterol management. “The main difference is the route of administration, with one being an oral medication and the other an injectable therapy. Both are intended to help lower LDL cholesterol in appropriate patients. The choice of treatment should be based on clinical need, treatment goals and patient preference, as determined by the treating physician,” he says.

Dr Khullar says, “In the near term, the route to Indian patients is Merck, or MSD as it is known here, bringing Lipfendra through CDSCO (Central Drugs Standard Control Organisation) approval. That process is not automatic and often requires local bridging data. The likely commercial pattern is the one we have already seen in this therapeutic space – Amgen partnered with Dr Reddy’s Laboratories to market Repatha in India, and inclisiran has since come in through a similar arrangement. A multinational-plus-Indian-partner model is the most probable path.”

Dr Kler is optimistic about a version of this pill being produced indigenously. “I would always prefer Indian pharmaceutical companies to make these drugs here because that will make them more affordable. Today, the Indian pharmaceutical industry has the capability to develop and manufacture these medicines. I have no doubt that we should encourage Indian companies to produce these drugs in the country,” he says.

Dr Kler explains, “As far as patient acceptability is concerned, it depends on two factors. One is the ease of taking the medicine. Since this is a tablet, patients will accept it more readily than an injectable drug. The second factor is the cost. The price of the drug will matter a lot, especially for Indian patients. If the cost is reasonable and it is available as an oral medication, then certainly many patients will switch from injections to this drug.”

“Removing the needle solves one barrier. It does not solve the price barrier, and we should not let the convenience story obscure that,” says Dr Khullar. He also caveats, “This drug is not a solution to India’s cholesterol problem. It is a very good tool for the subset of patients who are already diagnosed, already on maximum tolerated statin therapy, and still above target. That is a meaningful group and currently a badly served one. The much larger problem – that most Indians with high cholesterol have never had a lipid profile done – is not something a new molecule solves.”

“Daily tablets are generally easier to incorporate into routine life than periodic injections. By removing the barrier associated with injectable therapy, an oral formulation may encourage better treatment adherence,” explains Dr Malik. “Since its cholesterol-lowering efficacy appears comparable to injectable PCSK9 inhibitors, the key advantage lies in convenience, which often translates into better long-term cholesterol control.”

Abating crisis

First of all, we must ensure that every individual gets a lipid profile done by the age of 20-25 years. We should start early rather than late, says Dr Kler. “In India, many people do not know their cholesterol or LDL levels until they suffer a heart attack. Annual health check-ups should begin at a young age so that we know a person’s lipid profile early and can take appropriate action.”

Echoing early screenings, advised by the Lipid Association of India between ages 18 to 20, Dr Khullar adds, “Treat to target, not treat and forget. Prescribing a statin is not the same as controlling cholesterol. A very large proportion of Indian patients on lipid-lowering therapy never have a follow-up lipid profile. We should be starting combination therapy upfront in high-risk patients rather than slowly escalating doses over years.”

Dr Malik says that India needs greater public awareness about cholesterol, along with “routine screening of high-risk individuals, timely diagnosis, and evidence-based treatment. Improving access to effective therapies, while encouraging healthy eating, regular physical activity, tobacco cessation, and weight management, will be essential to reducing the country’s growing burden of cardiovascular disease.”

Disclaimer: The information in this article is for educational and informational purposes only and is not intended as medical advice. While the author has incorporated expert medical guidance while producing the story and ensured full authentic information is provided to the reader, you should always seek the advice of a qualified healthcare provider regarding a medical condition or treatment. Never disregard professional medical advice or delay in seeking it because of something you have read here.