The US Centers for Medicare & Medicaid Services (CMS) announced that coverage of Biogen’s Aduhlem (aducanumab) – and other US Food and Drug Administration (FDA) approved anti-amyloid monoclonal antibodies for Alzheimer’s disease – will be limited exclusively to participants in qualified clinical trials.

CMS’ decision is preliminary, with a final decision due to be made in April 2022, following a 30-day period for public comment. For Aduhelm and similar therapies, the policy will set national guidance on how Medicare treats these particular drugs in the future.

Six months after the FDA granted Aduhelm accelerated approval, CMS has requested further evidence ahead of any payment for the drug, priced at $28,000 a year.
The drug was given clearance on the strict condition that Biogen conducted an additional trial to confirm the benefits of using Aduhelm. However, regulators said at the time that the top priority was to provide patients with access to a potentially life-changing drug.

CMS officials made it clear that the risk-over-benefit needed significant consideration for patients receiving the treatment. Following a thorough analysis, CMS discovered that although Aduhelm had the potential to help patients with Alzheimer’s disease, the drug also had the potential to cause harm.

The officials also added that trials must be demographically representative of Medicare patients suffering with Alzheimer’s, given that the disease disproportionately affects people of colour in the US.

AstraZeneca and Daiichi Sankyo’s jointly developed drug, Enhertu (trastuzumab deruxtecan), has received a notification of acceptance of the supplemental Biologics License Application (sBLA), alongside a grant for priority review.

The drug is an HER2-directed antibody drug conjugate (ADC) and is a treatment option for adult patients in the US living with unresectable or metastatic breast HER2-positive breast cancer who have received a prior anti-HER2-formulated regimen.

Recorded as being the most common cancer worldwide, breast cancer-related deaths have accounted for nearly 685,000 deaths globally. For patients diagnosed with HER2-positive metastatic breast cancer, initial treatment with trastuzumab and a taxane does not prevent disease progression.

The announcement allows for further available treatment options for patients in order to delay disease progression and support survival.

During the DESTINY-Breast03 trial, Enhertu demonstrated a 72% reduction in the risk of death or disease progression in patients with HER2-positive unresectable metastatic breast cancer which was previously treated with trastuzumab and a taxane.

The drug is receiving further evaluation in a comprehensive clinical development programme to assess the safety and effectiveness across various HER2 cancers, including lung, breast, gastric and colorectal cancers.

The sBLA is currently under review by two FDA programmes, the Real-Time Oncology Review (RTOR) and Project Orbis, which are designed to provide effective cancer treatments to patients as early as possible.

Roche has announced that the US Food and Drug Administration (FDA) has approved a priority review of a supplemental new drug application (sNDA) for the use of Evrysdi in treating presymptomatic babies diagnosed with spinal muscular atrophy (SMA) under two months of age.

SMA is chronic, progressive neuromuscular disease, affecting an estimated one in 10,000 babies as a leading genetic cause of infant mortality. Depending on the patient’s form of SMA, physical strength, walking, eating or breathing can be impacted significantly or is lost altogether.

The FDA assessed the interim data submission from the RAINBOWFISH study, which demonstrated that the majority of presymptomatic babies who were administered Evrysdi achieved key milestones including sitting, walking, standing and a maintained ability to swallow having undergone 12 months of treatment.

The initial interim data gathered from the RAINBOWFISH study – presented at the World Muscle Society (WMS) Virtual Congress 2021 – provided findings that demonstrated all of the babies (5/5) involved in the interim efficacy analysis maintained the ability to swallow.

The study also showed that all of the babies were able to feed exclusively orally following 12 months of treatment, while 80% treated with Evrysdi for at least 12 months met key milestones such as standing and walking independently according to guidelines sets by the World Health Organization (WHO) windows for healthy children.

The European Commission (EC), European Medicines Agency (EMA) and EU regulators from The Heads of Medicines Agencies have set out proposals in a new initiative: Accelerating Clinical Trials in the EU (ACT EU).

The initiative aims to position the EU Member States as a collective international clinical research ‘focal point’.

The ACT EU document details objectives, priorities and governance strategies, involving measures to allow for a greater implementation of clinical trial designs. The aim of the initiative is for ACT EU to contribute to providing the Network strategy to 2025, alongside the Commission Pharmaceutical Strategy and the European Medicines Regulatory Network (EMRN) strategy in its goals to ‘foster innovation in clinical trials’.
Part of the EMA’s plan is to collaborate with other EU regulators to produce guidance on innovative trial designs, involving AI and machine ingenuity.

The paper recognises the impact of the COVID-19 pandemic as a major global event that has further highlighted the ‘disharmony of regulatory requirements between Member States’ and the consequential complications surrounding ‘the submission of multistate trial applications’, while many are still processed at a national level.

The initiative will further develop the EU’s 2014 Clinical Trials Regulation (CTR) in line with the launch of the Clinical Trials Information System (CTIS) – following a delayed launch in 2018 – which will begin at the end of January 2022.

Sanofi Genzyme and Regeneron Pharmaceuticals shared positive results from the second phase 3 for Dupixent (dupilumab), a medicine the companies collaboratively developed to target chronic inflammatory skin diseases.

Dupixent is a treatment that to date has been studied across 60 clinical trials involving over 10,000 patients living with various chronic conditions, specifically those suffering with type 2 inflammation.

Patients living with prurigo nodularis have reported the negative impact it can have on day-to-day living. A common prescription for the condition is a course of highly potent topical steroids which carry certain safety risks if used for a long-term treatment period.
The second phase 3 trial evaluating Dupixent involved adult patients diagnosed with uncontrolled prurigo nodularis – a chronic type 2 inflammatory skin disease – and met its primary and secondary endpoints.

These patients have either been ineligible for topical prescription therapies or have found that topical therapies have provided inadequate control for their condition. Throughout the 24-week trial period, patients were administered Dupixent or placebo every two weeks, with or without topical treatments.

The results demonstrated that the treatment significantly reduced skin lesions and itching versus placebo treatment at 24 weeks. The positive results back up those gathered from the data previously reported from the phase 3 PRIME2 trial and will be submitted for regulatory approval worldwide during the first half of 2022.