Sanofi seeks to launch Hemophilia A drug this year — CNBC

FRANCE — French drugmaker Sanofi SA plans to launch a drug for rare bleeding disorder hemophilia A this year, Chief Executive Officer Paul Hudson told CNBC in an interview.

The U.S. Food and Drug Administration last year accepted the marketing application for the therapy, which is being developed in collaboration with Swedish drugmaker Sobi, and is expected to decide on an approval status by Feb. 28.

Hemophilia A is an inherited bleeding disorder in which the blood does not clot normally. About 400 babies are born with this condition every year, although the exact number of people affected is not known, according to government data.

BioMarin’s valoctocogene roxaparvovec is already available in Europe as Roctavian for hemophilia A, but the biotech is still waiting for FDA approval.

The biotech is hoping that new Phase III data, which is expected in early 2023, will improve its regulatory chances.

The ongoing GENEr8-3 study by BioMarin uses a primary endpoint of change in factor VIII activity, testing valoctocogene roxaparvovec alongside corticosteroid use.

Meanwhile, Pfizer’s PF-06838435 is competing with CSL’s Hemgenix (etranacogene dezaparvovec) for the distinction of being the only other approved therapy for hemophilia B.

Hemophilia B is a less common form of the same blood disorder, caused by deficiencies in factor IX.

Pfizer intends to present key additional data from the Phase III BENEGENE-2 study (NCT03861273) of PF-06838435 in adult males with severe hemophilia B at a scientific conference in early 2023.

Pfizer announced in late December that BENEGENE-2 had achieved its primary endpoint of non-inferiority and superiority in the annualized bleeding rate (ABR), which experts describe as a validated marker of hemophilia activity.

Sanofi establishes rebate program after Pfizer

Meanwhile, Sanofi has followed in the footsteps of Pfizer by establishing a rebate program for blood disorder treatment, Cablivi, promising medical institutions a potential refund if inpatient treatment fails.

In order to qualify, patients must receive the drug in a hospital setting, and Sanofi will only cover the cost of up to six doses for those who do not respond, or up to 12 doses for those who worsen while on treatment.

Cablivi became the first drug approved in the United States in 2019 for acquired thrombotic thrombocytopenic purpura (aTTP).

Sanofi acquired the drug, also known as caplacizumab, in its US$4.8 billion Ablynx acquisition and received quick approval in Europe.

Patients with aTTP develop blood clots in their small arteries, which can cause strokes, brain damage, and death.

Sanofi set the initial list price for each course at US$270,000, generating €164 million (US$177 million) in revenue in 2021.

According to the company’s most recent earnings report, sales increased to €52 million (US$56 million) in Q3. Sanofi confirmed on Friday that the current wholesale acquisition cost per vial is US$7,925.

To make the cost more bearable, the company is now offering refunds of up to the full wholesale acquisition cost (WAC) or actual acquisition cost, whichever is less.

To qualify, the drug must be administered according to the label and discontinued by a healthcare provider.

Pfizer also launched similar warranty programs for its drugs Xalkori and Panzyga, though those programs reimburse patients rather than hospitals.

Panzyga was approved in 2018 to treat adults with primary immunodeficiency (PI) and chronic immune thrombocytopenia (cITP).

It later received approval for chronic inflammatory demyelinating polyneuropathy (CIDP), a condition characterized by arm or leg weakness, tingling or numbness, and a loss of deep tendon reflexes.

Pfizer has announced that it will reimburse certain CIPD patients for their out-of-pocket expenses for the first four treatments if they are forced to discontinue treatment for “clinical reasons,” up to a maximum of US$16,500 per treatment or US$50,000 total.

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