TOP Pharma & Biotech News #?? October Edition
In the October newsletter, explore the EMA’s EU Strategy to 2028, focusing on innovation and regulatory improvements, learn about the FDA’s approval of a groundbreaking treatment for Hemophilia A and B, and discover the UK’s world-first regulatory framework for personalized medicine manufacturing, aimed at enhancing patient care and access globally.
The European Medicines Agency (EMA) and the Heads of Medicines Agencies (HMA) have published a draft joint EU network strategy for 2028, now open for an eight-week public consultation.
This updated strategy replaces the original five-year plan (EMANS 2025), which covered 2021 to 2025. It aims to guide the future direction of the network, focusing on:
The EMA highlighted that technological advances like AI are transforming medicine development and regulation at a faster pace than expected, prompting the strategy’s revision. Recent developments, such as the creation of the Directorate-General for Health Emergency Preparedness and Response (DG HERA) to enhance crisis preparedness, and the biggest reform of EU pharmaceutical legislation in decades, further underscore the need for change.
EMA Executive Director Emer Cooke emphasized that the overarching theme of the revised strategy is rapid, somewhat unpredictable change, filled with promise. It aims to equip the network to meet future challenges, including responding to public health emergencies and threats like AMR.
The final strategy is expected to be adopted by March 2025. Feedback on the draft can be submitted online until 30 November 2024.
Source: EPR
The U.S. Food and Drug Administration (FDA) has approved Hympavzi (marstacimab-hncq) for routine prophylaxis, aimed at preventing or reducing the frequency of bleeding episodes in adult and pediatric patients aged 12 and older with hemophilia A or B, without factor VIII or IX inhibitors (neutralizing antibodies). According to Ann Farrell, M.D., Director of the Division of Non-Malignant Hematology at the FDA, the approval provides patients with hemophilia a new treatment option that works differently from existing therapies. This innovative drug targets a specific protein in the blood clotting process, making it the first of its kind. Dr. Farrell emphasized that this approval reflects the FDA’s commitment to advancing the development of new, effective therapies that are both safe and innovative.
Hemophilia A and B are genetic disorders that result from a deficiency or dysfunction of coagulation factors VIII (FVIII) or IX (FIX), respectively. Individuals with these conditions have difficulty clotting blood properly and may bleed for a longer time than normal after an injury or surgery. They are also at risk for spontaneous bleeding in muscles, joints, or organs, which can be life-threatening. Traditionally, these bleeding episodes are treated with on-demand, episodic therapies or through prophylaxis using products that contain FVIII or FIX, or through products that mimic clotting factors.
Hympavzi introduces a different approach to treatment by not replacing missing clotting factors but by targeting and reducing the activity of a natural anticoagulant protein called tissue factor pathway inhibitor (TFPI). This mechanism increases the generation of thrombin, a key enzyme involved in blood clotting. By enhancing thrombin production, Hympavzi is expected to lower the frequency and severity of bleeding episodes in hemophilia patients, offering a promising new approach to managing this condition.
The approval of Hympavzi is supported by data from an open-label, multi-center clinical study that included 116 male patients, both adults and children, with severe forms of hemophilia A or B, all of whom lacked inhibitors. During the first six months of the study, patients were treated with traditional clotting factor replacement, either on-demand (33 patients) or prophylactically (83 patients). After this period, all patients transitioned to 12 months of prophylactic treatment with Hympavzi. The study’s primary measure of efficacy was the annualized bleeding rate of treated bleeding episodes. In patients who had been using on-demand factor replacement therapy, the annualized bleeding rate was initially estimated at 38. After transitioning to Hympavzi, this rate dropped to 3.2, demonstrating a substantial improvement. For those receiving prophylactic factor replacement therapy, the bleeding rate shifted from 7.85 in the first six months to 5.08 during the subsequent 12 months on Hympavzi, showing that the new drug provided similar outcomes to established prophylactic treatments.
However, as with any treatment, Hympavzi comes with certain warnings and precautions. Patients taking the drug may be at risk for thromboembolic events (blood clots), hypersensitivity reactions, and embryofetal toxicity. It is important for healthcare providers to monitor for these potential risks during treatment. The most common side effects reported by patients during the study included reactions at the injection site, headaches, and itching (pruritis).
The FDA also granted Hympavzi Orphan Drug designation for this particular application, which helps support the development of treatments for rare diseases. This special designation provides certain incentives for the drug's development and approval process. The approval of Hympavzi was awarded to Pfizer Inc., marking another significant step forward in the treatment landscape for hemophilia patients.
Source: WORLD PHARMA NEWS
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The Medicines and Healthcare products Regulatory Agency (MHRA) has announced that a new regulatory framework could become law in the UK by next year.
The UK is poised to be the first country globally to introduce a new regulatory system for innovative medical products, particularly those that are manufactured at or near the point of patient care. This groundbreaking regulation will allow patients to gain quicker access to highly personalized treatments and medicines that have very short shelf lives. Examples include advanced therapies such as cell and gene treatments, which must be produced close to the individual patient to ensure safe and timely development.
One of the major advantages of this new regulation is that treatments can now be produced in small, portable manufacturing units. These units can be located near patients who are unable to travel, especially those with severe health conditions. By bringing the manufacturing process closer to the point of care, the burden on hospitals for delivering such treatments will also be significantly reduced.
Ian Rees, the Point of Care lead at MHRA, stated, “This regulation will enable new and innovative methods for manufacturing medicines closer to the patients who need them, while ensuring the highest standards of quality, safety, and efficacy are maintained.”
The framework was first proposed by the MHRA in January 2023, following a consultation held in 2021, where stakeholders showed overwhelming support for a regulatory system focused on point-of-care and modular manufacturing. This new approach is seen as critical to advancing healthcare in the UK and improving patient outcomes.
Furthermore, the regulatory framework is expected to increase the attractiveness of the UK as a key destination for launching new life-saving medicines and treatments. Ian Rees added that this move positions the UK at the forefront of innovative medical care.
To assist with the implementation of this regulation, the MHRA plans to release detailed guidance before the legislation takes effect. The new rules are anticipated to become law by the summer of 2025.
Source: EPR
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