Sanofi gets FDA nod for long-acting haemophilia A drug

Sanofi and Sobi’s haemophilia partnership has been under competitive pressure from new therapies like Roche’s antibody Hemlibra, and are hoping that a newly-approved long-acting drug can revitalise the franchise.

The FDA has cleared their once-weekly Factor VIII replacement therapy Altuviiio (efanesoctocog alfa) for adults and children with haemophilia A, giving Sanofi and Sobi a successor to well-established therapy Eloctate (efmoroctocog alfa), which is dosed every four days.

The new drug has been approved both for on-demand control of bleeding episodes in haemophilia A, as well as for routine prophylaxis, which will account for the bulk of the drug’s use.

Eloctate has been very successful for the duo, but has been hit hard by competition in the market, with 2022 sales booked by Sanofi declining 6% to €580 million ($614 million), as Hemlibra (eculizumab) continued to grow at a fast pace to reach around $4 billion.

Altuviiio – formerly known as BIVV001 – is Sanofi and Sobi’s main chance of clawing back market share and, according to analysts at Barclays, could do just that, with peak sales estimated to reach around €2.3 billion.

Sanofi has said it will price the drug at the same level of Eloctate, which is estimated to cost upwards of $275,000 per year in the US, depending on the weight of the patient, before discounts or rebates. That will make it a challenge to other factor therapies on the market from Bayer, Novo Nordisk, and Takeda’s Shire subsidiary.

The phase 3 XTEND-1 trial of Altuviiio in 159 subjects aged 12 or over showed that the drug restored Factor VIII levels to near-normal with once-weekly dosing, and was better at reducing the annualised rate of bleeding episodes than patients’ previous therapies. It is due to be filed for approval in Europe in the latter half of this year.

All told, there was a 77% reduction in ABR versus prior therapy to from 2.96 to 0.69 mean ABR in the study.

Sanofi's chief executive, Paul Hudson, said the approval means that patients and physicians can now “reimagine living with haemophilia”, as Altuviiio is the first drug to offer “powerful bleed protection” with a once-weekly dose.

Earlier this month, Hudson told investors that he expects the new drug to capture market share “both from factor and nonfactor therapies”, although analysts think older replacement therapies will be most affected, with Hemlibra largely unaffected - at least, in Altuviiio’s initial rollout.

“With Altuviiio, you’re allowing patients to be at near-normal factor levels for the majority of the week,” according to Bill Sibold – head of the Sanofi Genzyme unit, which will be responsible for marketing the new drug – who noted this is the first time that has been been offered to patients.

“We’re going to be spending a lot of time and are spending a lot of time in the community, educating what is possible now – possible to return somebody from experienced bleeds or at a high risk for bleeds to bringing them to an almost near-normal state.”

Sanofi acquired the drug when it bought Bioverativ in 2018 for $11.6 billion, and the company has previously said it thinks it has the potential to become a $2 billion-a-year blockbuster.