Prothena Corporation – a company with a broad portfolio of investigational therapeutics built on protein dysregulation expertise – has announced that the US Food and Drug Administration (FDA) has granted fast-track designation for its anti-amyloid beta antibody therapy, PRX012.

The potential ‘best-in-class’ treatment is currently being investigated in a phase 1 clinical study for the treatment of Alzheimer’s disease, a condition that, with greater global age expectation, is exponentially increasing throughout the world.

The fast-track programme is designed to accelerate the development and review of drugs intended to treat serious conditions, by incorporating compelling evidence which demonstrates the potential to address unmet medical needs.

Preclinical data has revealed that binding PRX012 to beta amyloid plaques and oligomers with high avidity allows effective Aβ plaque occupancy at relatively lower dose ranges – optimal for subcutaneous delivery.

There was also a demonstrable clearance of both pyroglutamate modified and unmodified Aβ plaque in brain tissue, using quantities of PRX012 estimated to be clinically achievable in the central nervous system with subcutaneous delivery.

Approximately 50 million people worldwide are estimated to be living with Alzheimer’s disease or other dementias, with Alzheimer’s disease being the most common neurodegenerative disorder.

The European Commission (EC) has approved Novartis’ CAR-T cell therapy, Kymriah (tisagenlecleucel), to treat adults with relapsed or refractory follicular lymphoma, an advanced blood cancer, after two or more previous therapies.

The EC’s decision follows a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) issued in March, making Kymriah the first CAR-T cell therapy of its kind approved in the EU.

The approval applies to all 27 EU member states, as well as Iceland, Norway and Liechtenstein.

Approval was based on the ELARA global phase 2 study, which demonstrated that 86% of patients who were treated with Kymriah had a response, including 69% who had a complete response.

In addition to refractory follicular lymphoma, Kymriah is also approved to treat paediatric and young adults who are 25 years of age or younger with B-cell acute lymphoblastic leukaemia (ALL) that is refractory, in relapse post-transplant, or in a second or later relapse.

The treatment has also been approved for adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL), following two or more systemic therapies.

Pfizer and Valneva have announced positive phase 2 paediatric results for their Lyme disease vaccine candidate, VLA15.

VLA15 is currently the only Lyme disease vaccine candidate in clinical development. The vaccine uses a mechanism that targets the outer surface protein A of Borrelia burgdorferi, the bacteria transmitted from Ixodes ticks.

Lyme disease is a systemic infection transmitted to people by infected Ixodes ticks carrying Borrelia burgdorferi bacteria. It is estimated to affect around 476,000 people in the US and 130,000 people in Europe each year.

The phase 2 randomised study is the first clinical study using VLA15 with an enrolled paediatric group – those aged five to 17 years. It compared the immunogenicity and safety of the vaccine after administration of two or three primary doses in groups aged five to 11, 12 to 17 and 18 to 65 years.

Paediatric patients who received VLA15 – either as two or three doses – showed that VLA15 offered more immunity than in adults. The data builds on the strong immunogenicity profile already reported for adults (aged 18 to 65 years) in February 2022.

The safety and tolerability of the vaccine given to the five to 17-year age group was similar to the earlier reported profile in adults, while no vaccine-related serious adverse events were recorded.

Roche has released three-year data from its FIREFISH study, including one-year data from the open label extension.

FIREFISH evaluated the efficacy and safety of Evrysdi (risdiplam) in babies aged one to seven months at the time of enrolment with type 1 spinal muscular atrophy (SMA). The study was in two parts, with Part 1 confirming the dosage and Part 2 evaluating the efficacy and safety of that dosage.

The analysis reinforced the long-term efficacy and safety of Evrysdi, with data also showing an estimated 91% of babies treated with the drug were alive after three years of treatment.

Babies continued to improve or maintain motor functions, notably, the ability to swallow, sit without support, stand with support and walk while holding on, following two and three years of treatment. Without the treatment, children with type 1 SMA are not able to sit without support.

The study also showed overall continued reductions in serious adverse events and hospitalisations over time.

Evrysdi is designed to treat SMA by increasing and sustaining the production of the survival motor neuron (SMN) protein in the central nervous system and peripheral tissues. SMN protein is found throughout the body and is critical for maintaining healthy motor neurons and movement.

NHS doctors will now be able to spot a rare form of eye cancer among babies in the womb, following the roll-out of a new testing procedure across the NHS.

The new NHS test – developed at Birmingham Women’s and Children’s NHS Foundation Trust – identifies those at risk of developing retinoblastoma.

Symptoms of the condition are often hard to detect and a diagnosis can normally be made only once the tumour has progressed, by which time the eye cannot be saved.

This new non-invasive test can detect changes in the genes and is likely to identify around 50 infants with retinoblastoma every year. Non-invasive prenatal diagnosis also means parents can be informed early in pregnancy if their child is at risk.

A blood sample is taken from the mother before birth and analysed for mutations, which can determine with almost 100% accuracy if the baby will develop retinoblastoma.

Vital treatment can then start on the affected eye as soon as the baby is born, while doctors can also closely monitor the other eye. In addition, the test can predict if the disease might develop in siblings and therefore will be offered to those who have a confirmed case of retinoblastoma in the family.