Advanced Therapies

Christopher Anzalone is spearheading Arrowhead Pharmaceuticals' transformation from a development-focused to a commercial-stage biotech. The CEO touts an “almost plug and play” platform, a derisked development strategy and a busy pipeline with vast potential.

It is “absolutely fundamental” that manufacturers of cell and gene therapies interact with inspectors from regulatory agencies to understand their expectations around good manufacturing practice, a compliance consultant at CDMO eXmoor pharma says.

Nine new entries targeting a wide range of unmet medical needs. Several EU marketing approvals and two withdrawals. And an accelerated assessment tool that was seldom used. These were among the activities noted as part of the European Medicines Agency’s priority medicines scheme during the first half of 2024.

After a quiet July, August and September are shaping up to be busy on the approvals front. Already this month, the US FDA has approved Adaptimmune’s Teclera for synovial sarcoma, Phathom’s Voquezna for gastro-esophageal reflux disease and Servier’s Voranigo for gliomas. Here, Scrip takes a look at ten other approvals for novel products in the offing for the third quarter.    

In June, the US Supreme Court reversed the Chevron doctrine, a long-standing precedent requiring courts to defer to regulatory agencies when statutory language was ambiguous. But will that decision prevent the FDA’s final rule on laboratory developed tests from taking effect? A legal expert weighs in.

AstraZeneca’s sipavibart for preventing COVID-19 in the highly vulnerable population of immunocompromised patients is among the latest drugs that the European Medicines Agency has started to review for potential pan-EU marketing authorization.

Increased R&D spending, program acquisitions and a soaring headcount – Regeneron has seen significant growth over the past 5 years. The company is focused on marrying its antibody technologies with its expanding cell and gene therapy pipeline. 

Two new pieces of guidance, from the EMA and the HTA coordination group respectively, should help companies understand which drugs must undergo an EU-wide joint clinical assessment and how to notify the relevant authorities about them.

The gene therapy is now approved for all ambulatory boys with Duchenne muscular dystrophy ages 4 and older. The revised label also includes accelerated approval for non-ambulatory patients.

A first big pharma partnership for Ascidian, headed up by ex-Biogen research head Michael Ehlers, as the Swiss firm expands its gene editing and neuroscience efforts.

Breathomics, UTI diagnosis and advanced wound healing innovations were among the center stage technologies at BioWales in London 2024. Hear from CEO John McKinley on Imspex Diagnostics' plans in embedded podcast.  

The latest meeting of the European Medicines Agency’s human medicines committee, the CHMP, is taking place this week. Decisions are expected for 13 drug marketing applications, there will be a number of oral explanation meetings, and activity is expected relating to a drug at the center of a case involving the European Commission’s rejection of an EMA recommendation.

Breathomics, UTI diagnosis and advanced wound healing innovations were among the center stage technologies at BioWales in London 2024.

EU-level action on market access could include a “solidarity fund” for ultra-rare disease treatments and a new framework for managed entry agreements.

Advances in synthetic biology are moving biopharma closer to a world where treatments can be tailored while remaining cost-effective.  

The US FDA's controversial final rule places laboratory developed tests under same regulatory purview as other in vitro diagnostics. While the agency opted to include an exemption for LDTs already on the market, it did not provide an exemption for tests developed in academic medical centers.

After much anticipation, the US FDA is set to publish its controversial final rule for regulating laboratory developed tests, which places the tests under the same regulatory purview as other in vitro diagnostics. While the agency opted to include an exemption for LDTs already on the market, it did not provide an exemption for tests developed in academic medical centers.

At the recent Precision Med-Tri Con conference, laboratory experts traded views on the expansion of at-home testing for disease diagnosis and personalized health insights. While strong consumer demand spells opportunity, there are significant concerns about the accuracy and reliability of home-testing platforms, misuse, accessibility, and lack of health literacy.

Bristol Myers Squibb’s head of major markets, Monica Shaw, wants to improve patients’ access to lifesaving therapies. And the group has several new products ready to roll.    

During a recent hearing of the US House Energy and Commerce Health Subcommittee, lawmakers discussed the best approach to regulating laboratory developed tests (LDTs) with a panel of experts representing clinical labs. At issue was the FDA’s proposed rule that places LDTs under the same regulatory purview as other in vitro diagnostics, which would require medical facilities to receive agency approval for the LDTs they develop in their own labs.