Biden administration commits to improving gene therapy accessibility
In December, history was made with the FDA approving two groundbreaking therapies on the same day: Vertex and CRISPR Therapeutics' Exa-cel, a CRISPR/Cas9 genome-edited cell therapy for sickle cell disease (SCD), and bluebird bio's gene therapy, Lyfgenia, targeting the underlying cause of SCD.
Exa-cel (Casgevy) offers a potential one-time transformative therapy for SCD patients aged 12 and older with recurrent vaso-occlusive crises (VOCs). Lyfgenia, a gene therapy, aims to resolve VOCs and treats SCD by adding a functional β-globin gene to patients' hematopoietic stem cells.
However, the high cost of these therapies raised concerns about accessibility. In response, the Biden-Harris Administration recently designated SCD as the initial focus of the Cell and Gene Therapy (CGT) Access Model. Set to launch in 2025, this model, aligned with efforts to reduce prescription drug costs, seeks to improve health outcomes, increase access and lower costs for vulnerable populations.
Medicaid recipients, constituting 50-60% of SCD patients, incur nearly $3 billion in annual health system costs related to SCD episodes. The CGT Access Model involves CMS negotiating outcomes-based agreements with manufacturers, tying pricing to enhanced health outcomes for Medicaid recipients.
For its part, bluebird bio revealed in financial updates earlier this week that it had secured "access and reimbursement strategy, for Lyfgenia, "driving favorable coverage landscape," and "continues to engage with Center for Medicare and Medicaid Innovation on its Cell and Gene Therapy Access Model, which is anticipated to be implemented in 2025."
The announcement brings a huge win for patients, signaling a potential breakthrough in accessibility to transformative therapies and fostering collaboration between regulators and the industry to address pressing health care challenges. — Andrea Corona
The Aduhelm exit
This week, Biogen, in a plan to "reprioritize its resources in Alzheimer’s disease," announced that it will discontinue its controversial Aduhelm program and terminate the post-marketing confirmatory study.
In general, it’s not a good look when a company halts its post-approval study — a study that exists to prove the drug’s benefit in the real world. One might conclude here that company in fact knows the drug isn’t going to prove effective.
But I’m not here (nor am I qualified) to assess whether or not Aduhelm works. And certainly enough analysis already exists over the FDA’s approval process, including a 46-page report detailing an 18-month Congressional investigation.
But one thing Biogen’s CEO Christopher Viehbacher said in the press statement did resonate: “When searching for new medicines, one breakthrough can be the foundation that triggers future medicines to be developed. Aduhelm was that groundbreaking discovery that paved the way for a new class of drugs and reinvigorated investments in the field.”
In all fairness, that is the crux of how advancements are made in the pharma industry. Rarely is the first drug on the market the final say in treatment. Post-marketing studies take time and money and it’s reasonable to conceive there would be advancements in the field by the time Aduhelm could apply for FDA traditional approval.
What’s notable here is that Biogen’s decision gives the full rights to aducanumab back to the company that discovered the drug — Switzerland-based Neurimmune.
And it doesn’t seem as though Neurimmune is giving up on the drug. “We are excited to lead the future development of aducanumab,” said Fabian Buller, Neurimmune’s chief business officer, in a press release. “We aim to develop aducanumab-based therapeutics for early intervention with a focus on subcutaneous administrations, and will provide updates on our progress in due course.”
I think it’s going to be interesting to see what the company does with the drug.
In terms of Biogen’s Aduhelm saga, I believe it was P.T. Barnum who once said, “There's no such thing as bad publicity.” At the very least, the drug got the world talking about a treatment space that has been bereft of options for decades — and hopefully this leads to new advancements for patients. — Karen Langhauser
