HAS restricted iptacopan to second-line use, anemic patients below 10 g/dL haemoglobin after six months on anti-C5 therapy.

France's Commission de la Transparence rated iptacopan (Fabhalta) ASMR III, a moderate improvement, in its 5 December 2024 economic opinion. That rating came with a restriction the FDA and MHRA labels do not carry: HAS positions iptacopan as a second-line option only, for adult patients with persistent hemolytic anemia, haemoglobin below 10 g/dL, after at least six months on a C5 complement inhibitor. Ravulizumab (Ultomiris) holds the opposite position: its SMR is rated important, and HAS considers it a first-line medicine for PNH, citing its every-eight-week dosing advantage over eculizumab's every-two-week schedule. Ravulizumab defends first place; iptacopan has to earn its way in from second line.

That asymmetry sets the CEPS price negotiation up differently for each drug. Iptacopan's published reimbursement price is €29,590.96 excluding tax per box of 56 tablets, negotiated against a target population the manufacturer itself estimated at only 270 patients nationally, a genuinely small commercial base for a specialty price point. Ravulizumab's published price runs €14,988.66 for the 1100mg vial and €4,087.82 for the 300mg vial, reflecting weight-based dosing across a first-line population that never had to argue past a prior-treatment gate. France's second-line restriction shrinks iptacopan's addressable population before a single euro of price negotiation happens.

France shrinks iptacopan's addressable population before a euro of pricing is negotiated.

Five questions this report answers:

Q1 - Why did HAS restrict iptacopan to second-line use when the US and UK labels don't?

Q2 - What does a target population of just 270 patients imply for the CEPS price negotiation?

Q3 - How does ravulizumab's first-line SMR-important position change the comparator argument for new entrants?

Q4 - What does the second-line restriction mean for iptacopan's addressable population and CEPS pricing?

Q5 - What dosing advantage did HAS cite to keep ravulizumab in the first-line position?

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Mike || Global Pharma Commercial Marketing Head

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