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When shortage biotherapies puts lives:
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Following repeated alerts on the risk of rupture of access to anti-PCSK9 drugs. For example, the association of patients suffering from family hypercholesterolemia (ANHET.F. For example, the National Association of Family Hypercholes and Lipoproteins (A)) and specialists invested in this potentially serious disease were received by the Minister of Health on July 16.
What is family hypercholesterolemia? – When shortage biotherapies puts lives
Family hypercholesterolemia, disease of genetic origin, is a hereditary form of excess cholesterol. Nevertheless, It causes. Consequently, from birth, a sometimes very high when shortage biotherapies puts lives rise in LDL cholesterol level (“bad cholesterol”), with a clearly increased cardiovascular risk.
Heterozygous family hypercholesterolemia, the most frequent, affects approximately 1 in 250 people, or nearly 300,000 people in France. Meanwhile, For its part. Moreover, the Homozygous family hypercholesterolemia, much rarer (1 person in 500,000), exposes to very severe complications from childhood: without treatment, this homozygous form (LDL cholesterol rate 6 to 8 times higher than normal) can lead to angina (angina of chest: lack of oxygen of the cardiac), a myocardial infarction or even death sudden before 30 years old, sometimes from childhood.
But the heterozygous form (LDL cholesterol level at least 2 times higher than normal) is also serious: around 50 % of unrealized men will make a cardiovascular accident before 50 years. Consequently, and 30 % of women before 60 years. Furthermore, These accidents can occur from the age of 20. For example, In the absence when shortage biotherapies puts lives of treatment. Furthermore, the state of arteries at 40 in these patients is comparable to that of a person of 80 years.
Family hypercholesterolemia remains largely overlooked: only 20 % of the persons concerned are today diagnosed.
What are anti-PCSK9?
About ten years ago. the arrival of anti-PCSK9 drugs (Alirocumab and Evolocumab) was announced as a revolution for people with family hypercholesterolemia. These are biotherapies intended for people with a high cholesterol level which does not decrease despite an optimized classic hypolipaning treatment (statins. / or ezetimibe), as in the event of family hypercholesterolemia, hypercholesterolemia resistant to statins or in people with recent history of myocardial infarction (non -stroke Hemorrhagic) and/or obliterating arteriopathy of the lower limbs (AOMI) symptomatic and which retain a very high cholesterol level despite, in particular statins with maximum tolerated doses. Among others.
Their mechanism? They target a protein. the PCSK9 which has a role in the when shortage biotherapies puts lives capacity of the liver to assimilate the LDL cholesterol. By inhibiting the binding of the PCSK9 with the LDL cholesterol receptor. they increase the density of the receptors on the surface of the liver cells, and thus decrease the circulating cholesterol LDL.
These expensive treatments, reserved for very specific situations, are subject to a request for prior agreement of health insurance. Their reimbursement in France was progressive from 2020-2021 and their prescription was extended in 2022. But the difficulties of access to these molecules remain considerable for specialists who have the authorization to prescribe them.
A (expensive) backtracking?
Lionel Ribes, president of Anhet.f. recalled on July 17 at a press conference that ” Many regions of France are no longer supplied with anti-PCSK9*, with the key to a foreseeable increase in hospitalizations of unsolved patients (…) “, recalling that” Neuro-Cardiovascular diseases are responsible for 140,000 deaths per year ». The anhet.f when shortage biotherapies puts lives receives daily calls for patients facing a double penalty: that of the disease. that of the impossibility of accessing a treatment however available in other countries of the European Union. Hence the mobilization of learned societies alongside the associations of patients and alliance rare diseases.
Professor Vincent Durlach, member of the scientific council of Anhet.F. explained that, for lack of anti-PCSK9 available, doctors could be forced to use LDL-APHERES to treat the patients most at risk in the neuro-cardiovascular level. LDL-APHERESE is an already old technique which makes it possible to mechanically eliminate the LDL-cholesterol of the blood. Comparable to a dialysis session. it specifically filters atherogenic lipoproteins (that is to say those which promote the formation of deposits in the arteries) and makes it possible to reduce their concentration in plasma by around 70 %. It is an expensive. invasive and restrictive method, generally reserved for the most serious when shortage biotherapies puts lives cases, when drugs are no longer enough. Where when they are not or more accessible …
Find out more: National Association of Family Hypercholesterols and Lipoproteins (A)
* ANSM alert in September 2024 and April 28, 2025. The Prime Minister launched on June 26 a flash mission on the price of medicines. one of the producing laboratories announced the reimbursement of its anti-PCSK9 biotherapy.
Source: Press release July 17, 2025 ANHET.F “High-high-Cardiovascular patient access to anti-PCSK9 patients. The call of the National Association of Family Hypercholesterols and Lipoproteins (A) heard by the Minister of Health and Access to Care ”
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