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Novartis eyes $1B sales with FDA nod for targeted sickle cell disease drug Adakveo

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Another candidate for Novartis’ future blockbuster membership is right here.

Friday, the Swiss drugmaker gained FDA approval for Adakveo, the primary targeted remedy to forestall the painful vaso-occlusive crises (VOCs) that come alongside with sickle cell disease. The nod got here weeks forward of schedule, even on high of a precedence evaluate doled out in July.

Novartis has priced the brand new drug, meant for sufferers 16 and older, at a wholesale acquisition value (WAC) of $2,357 per vial. As it’s prescribed based mostly on weight, most sufferers would take three or 4 vials a month. That would translate into WACs of $7,071, or $9,428, per 30 days.

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In Adakveo, Novartis is trying to fill an innovation void in sickle cell disease and ship billion-dollar peak sales; the drug is amongst a dozen potential blockbuster launches by 2021 CEO Vas Narasimhan has laid out.

“The biggest factor today that patients complain about, despite all the available standard of care, is VOC,” Andrew Cavey, Novartis’ world program head of benign hematology, informed FiercePharma forward of the approval. “And these are unpredictable, severe events that can cause life-threatening complications.”

Each VOC occasion impacts a affected person’s high quality of life, causes new organ harm and will increase the danger of dying, he stated.

RELATED: Novartis CEO touts 25 potential—and numerous—blockbuster launches. But is its portfolio too scattered?

Adakveo is designed to scale back VOCs by inhibiting the P-selectin protein, which performs a key position in how completely different cells inside blood vessels stick to at least one one other and trigger VOCs. Novartis acquired the drug in 2016 through its $665 million buyout of Selexys Pharmaceuticals.

In a section 2 examine dubbed Sustain, Adakveo confirmed it might lower the median annual price of VOCs by 45% in contrast with placebo, or 1.63 occasions versus 2.98. As many as 36% of sufferers on Adakveo didn’t expertise a VOC in any respect in the course of the 52-week trial, in comparison with 17% for the placebo arm. It additionally decreased the median days a affected person was hospitalized in a yr by 42%.

That final enchancment isn’t solely related for sufferers however might additionally place Adakveo nicely in reimbursement talks with payers, as a result of it truly lowers healthcare prices, Cavey stated.

When it involves the launch, Cavey stated Novartis can leverage its expertise in hematology, pointing to the truth that about half of its oncology portfolio falls in blood problems.

In reality, Novartis already has relationships with sickle cell disease sufferers and their clinicians through Exjade/Jadenu, which is accepted to deal with excessive ranges of iron on account of blood transfusions, he famous. Sickle cell disease sufferers may obtain blood transfusions to scale back signs.

RELATED: GlycoMimetics shares almost lower in half as Pfizer-partnered section 3 drug flops

Existing sickle cell disease therapies embrace Eammaus Life Sciences’ Endari, which is an oral powder formulation of amino acid L-glutamate designed to enhance the perform of hemoglobin, the blood’s oxygen transporter. Approved by the FDA in 2017 as the primary therapy for sickle cell disease in about 20 years, the drug hasn’t taken off, accumulating solely $17.3 million sales within the first 9 months of 2019.

Pfizer-partnered GlycoMimetics’ rivipansel, which additionally goals to dam the exercise of the selectin cell adhesion molecules, simply flopped a section 3 in hospitalized sickle cell disease sufferers within the midst of a VOC. It failed throughout the board on the first endpoint of readiness-for-discharge and a few key secondary endpoints.

There’s additionally the potential risk from gene remedy. Even although it might be years away, bluebird bio has started scientific testing of its EU-approved beta-thalassemia gene remedy Zynteglo (LentiGlobin) in extreme sickle cell disease.

RELATED: Reversing sickle cell disease with CRISPR-edited stem cells

Novartis, identified for its curiosity in novel gene and cell therapies, has itself invested within the space via a collaboration with Intellia Therapeutics. The pair is leveraging Intellia’s CRISPR-Cas9 gene-editing platform to tweak human hematopoietic stem cells.

By growing the manufacturing of fetal hemoglobin to switch the sickled crimson blood cells, the strategy holds promise to ameliorate the disease. The firms reported encouraging ex vivo leads to late 2017, and Cavey stated they hope to enter the clinic quickly.

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