A new class of cholesterol drugs will undergo a preliminary review this week from a Food and Drug Administration (FDA) advisory panel. The drugs, Amgen’s Evolocumab and Alirocumab from Sanofi SA and partner, Regeneron Pharmaceuticals, block a protein called PCSK9, which can prevent the liver from eliminating bad cholesterol. Early studies show the drugs to dramatically reduce LDL cholesterol, and if approved, would be the first innovation in cholesterol medicine since the introduction of statins in the late 1980s. Statins like Pfizer’s Lipitor and AstraZenica’s Crestor have dominated the market ever since, but some patients are unable to process those drugs in their system, or the medicines have not been effective at reducing cholesterol. Bill Sasiela, executive and program director at Regeneron says, “This is the next big quantum leap in being able to control cholesterol and potentially reduce the risk of heart attacks and strokes.” While statins like Crestor and Lipitor will likely continue to dominate the market regardless of the PCSK9-blockers’ pending approval, Goldman Sachs estimates that the drugs could generate over $10 billion worldwide annually due to the extremely high demand for cholesterol medications.
FDA approval is not inevitable, however, as several concerns exist about the effectiveness, cost, and the side-effects of the new drug class. Although testing has shown the drugs to facilitate a reduction in LDL, or “bad cholesterol,” company-sponsored outcome testing as to whether or not this reduction leads to significant improvement in heart and cardiovascular health will likely not be concluded until late 2016 or 2017. The FDA has generally assumed the connection between the two, but there is a slim possibility that the lack of a conclusive link could be a roadblock. More concerning to the FDA, however, will likely be the potential side-effects, which may include memory-impairment and delirium. The drug makers insist that they have found no heightened or significant risk of these side-effects in early testing, but have also said that more testing will need to be conducted. Other concerns from early testing include an imbalance in the number of cases of pancreatitis and serious kidney disorders as well as a possible increase in new onset diabetes in certain patients. FDA reviewer, Dr. Eileen Craig says however, that the latter problems could be adequately addressed in the drug’s labeling and proper monitoring by healthcare providers. The final potential roadblock to the drugs’ widespread use could be cost. Analysts at Sanford C. Bernstein & Co. estimate that the drugs could cost users over $1000 per month, which could cause a pushback form health plans and benefit managers. Jay Edelberg, director of the PCSK9 development team for Sanofi, says the public health benefits of reduced cardiovascular disease “will provide significant medical value.”
Given the potentially vast market size for anti-cholesterol drugs worldwide, the companies have been very aggressive in their push for FDA approval. Sanofi and Regeneron paid a $67.5 million voucher for the FDA to conduct the FDA review ahead of Amgen, bumping up the FDA’s decision deadline for late-July, a month earlier than Sanofi and Regeneron. Amgen, for their part, filed a patent suit last October against Sanofi and Regeneron in an attempt to prevent sales in the event of FDA approval. Both drug makers have also touted their respective dosing regimens as being superior to the other in marketing communications. Also, both companies have been using their drugs’ brand names already, a move that usually isn’t made until they garner FDA approval. Amgen’s drug will be called Repatha, countered by Sanofi and Regeneron’s, Praluent. Currently, both drugs come in injection form, with users capable of performing the injections at home. Pfizer, however, is also working on its own PCSK9 drug, which may come in pill and vaccine form, as well as an injection. Despite being late to the table on its own version of the new drugs, the Pfizer developer heading the company’s PCSK9 development, Geno Germano, still sees much room for opportunity. “Given the size and diversity of patients,” he says, “we think there is an opportunity to be competitive.” Although the FDA is not obligated to approve a drug that has been cleared by the review panel, it is usually a strong indicator of the process’s eventual success. If any or all of these drugs are approved, the efforts of these drug makers could offer a new world of possibilities for the prevention of heart disease.
Empire State Tribune – Bob Gore
MarketWatch – Jonathan D. Rockoff
Reuters – Toni Clark