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ICER Says Gene Therapies For Sickle Cell Likely To Improve Lives, But Points To Uncertainties

 
• By Alaric DeArment

The drug pricing watchdog said improvement to patients’ quality and length of life from lovo-cel and exa-cel were likely, but noted ongoing uncertainty around durability and toxicity concerns.

ICER released a draft evidence report about two gene therapies for sickle cell disease • Source: Shutterstock

With the US Food and Drug Administration likely to soon have two applications for gene therapies to treat sickle cell disease (SCD) on its plate, the Institute for Clinical and Economic Review (ICER) is assuming a price of $2m with potential outcomes-based reimbursement agreements for each while emphasizing the need to balance their potential to improve patient’s lives with ongoing uncertainty about their durability as well as concerns about adverse events, particularly from myeloablative conditioning.

The drug pricing watchdog released the draft evidence report on 12 April for the two therapies, bluebird bio’s lentiviral vector-based lovotibeglogene autotemcel (lovo-cel) and Vertex Pharmaceuticals Incorporated and CRISPR Therapeutics AG’s

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Bluebird, Private Equity Firms Look To Sweeten Buyout Deal

 
• By Alaric DeArment

Shareholders are being offered a flat fee of $5 per share as an alternative to $3 plus a contingent value right, which could be a more attractive option if they want cash up front.

Which Firms Are Most Exposed To Prasad’s Possible Accelerated Approvals Clampdown?

 
• By Andrew McConaghie

Vaccine and accelerated approvals are expected to face greater scrutiny under new CBER leader Vinay Prasad, but he has promised “no light switch change” to policy, giving some reassurance to companies whose share prices have been battered.

Abeona Sets $3.1m Price For Rare Skin Disorder Gene Therapy

 
• By Joseph Haas

Abeona plans to offer an outcomes-based payment model for Zevaskyn, which likely will see complementary use with Krystal’s Vyjuvek in recessive dystrophic epidermolysis bullosa patients.

Verve Moves Toward Lilly Opt-In For PCSK9 Program With Phase Ib Results

 
• By Alaric DeArment

The company posted positive initial results from the Heart-2 trial in HeFH and CAD, with strong LDL cholesterol lowering and a clean safety profile.

More from Advanced Therapies

Abeona Sets $3.1m Price For Rare Skin Disorder Gene Therapy

 
• By Joseph Haas

Abeona plans to offer an outcomes-based payment model for Zevaskyn, which likely will see complementary use with Krystal’s Vyjuvek in recessive dystrophic epidermolysis bullosa patients.

Gilead Pipeline Progress Offsets Oncology Sales Slowdown

 
• By Alaric DeArment

Sales of the company’s approved cancer therapies were weaker or down, but Trodelvy is likely to see a label expansion and cell therapies are making progress.

In Brief: BriaCell’s Breast Cancer Cell Therapy Boosts Survival In Phase II

 
• By Sushmita Panda

BriaCell announced its cell therapy Bria-IMT plus checkpoint inhibitors bested Gilead’s Trodelvy in a Phase II trial in HR+ breast cancer; a Phase III trial is enrolling.