Our weekly newsletter provides you with the latest industry news, developments and regulatory agency updates.

Regulatory Updates

MHRA guidance updates

  • MHRA Windsor Framework. On 11th October, the Medicines and Healthcare products Regulatory Agency (MHRA) added the September webinar to guidance on the Windsor Framework agreement on the supply of medicines in Northern Ireland. Click to view source

FDA guidance updates

  • Temporary Policies for Compounding Certain Parenteral Drug Products. On 11th October, the Food and Drug Administration (FDA) published final guidance describing the agency’s regulatory and enforcement priorities regarding the compounding of certain parenteral drug products by outsourcing facilities and by State-licensed pharmacies and Federal facilities that are not registered with the FDA as outsourcing facilities. Click to view source
  • CVM GFI #284 Using Relative Supersaturation to Support “Urinary Tract Health” Claims for Adult Maintenance Cat Food. On 15th October, final guidance was published with recommendations for how pet food manufacturers can use RSS methodology to substantiate labelling that an adult maintenance cat food supports urinary tract health by promoting a healthy mineral content in the urinary tract. Click to view source
  • Requests for Reconsideration at the Division Level Under GDUFA. On 16th October, the FDA announced the availability of final guidance to provide recommendations on the procedures for applicants of abbreviated new drug applications (ANDAs) who wish to pursue a request for reconsideration within the review discipline at the division level or original signatory authority. Click to view source

FDA to host Informed Consent webinar

On 8th November, the FDA will host a public webinar entitled “Informed Consent – More than Just Another Document to Sign?” The event will be hosted by the Office of the Chief Medical Officer’s Office of Clinical Policy and the Office of External Affairs’ Public Engagement Staff, both in the Office of the Commissioner, and will provide patients and researchers with an overview of the FDA’s expectations for informed consent. For further information, visit the FDA website.

EMA launches consultation on handling competing interests of scientific committee members and experts

On 10th October, the European Medicines Agency (EMA) announced the revision of its policy on handling of competing interests of scientific committee members and experts, aligning it with recent court rulings to ensure impartiality in assessments. The changes include increased restrictions across roles and groups on experts with current interests in products, a unified three-year cooling-off period for past industry involvement, and updated rules for medical device experts. Public consultation on the draft policy is open until the 10th of November 2024 with the final version by EMA’s Management Board expected by the end of the year.

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Industry Updates

First anti-tissue factor pathway inhibitor for the treatment of hemophilia A or B approved in the US

On 11th October, Pfizer Inc. announced the FDA approval of HYMPAVZI™ (marstacimab-hncq) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and paediatric patients 12 years of age and older with hemophilia A (congenital factor VIII deficiency) without factor VIII (FVIII) inhibitors, or hemophilia B (congenital factor IX deficiency) without factor IX (FIX) inhibitors. HYMPAVZI is the first and only anti-tissue factor pathway inhibitor (anti-TFPI) approved in the US for the treatment of hemophilia A or B and the first hemophilia medicine approved in the US to be administered via a pre-filled, auto-injector pen.

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Samsung Biologics launches high-concentration formulation platform to accelerate high-dose drug development

On 14th October, Samsung Biologics launched a new high-concentration formulation platform aimed at accelerating the development of high-dose drugs. S-HiConTM supports the formulation of biologics at higher concentrations, reducing the volume required for patient dosing, which can improve patient convenience and treatment efficiency. It is part of Samsung Biologics’ continued efforts to provide clients with innovative technologies that enable high-quality development.

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Lundbeck to acquire Longboard Pharmaceuticals, significantly enhancing its neuroscience pipeline

On 14th October, Lundbeck announced it has entered an agreement to acquire Longboard Pharmaceuticals, enhancing its neuroscience pipeline with a focus on treating Developmental and Epileptic Encephalopathies (DEEs). The transaction is anticipated to close in the fourth quarter of 2024, subject to regulatory clearances and other standard conditions.

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Alleo and Ubiquigent enter AI-driven partnership to advance DUB-focused drug discovery

On 15th October, Ubiquigent Limited announced a partnership with Alleo to develop new therapeutics for neurological diseases. Ubiquigent will use Alleo’s AI software, RubDUB, to enhance its search for novel deubiquitinase (DUB) modulators, including inhibitors and DUB-targeting PROTACs. In return, Alleo will leverage Ubiquigent’s expertise and platforms to progress its own DUB-focused precision neuroscience initiatives.

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FDA’s 2024 ARC program report highlights continued innovation in rare disease drug development

On 15th October, the FDA’s Center for Drug Evaluation and Research (CDER) released the 2024 Accelerating Rare Disease Cures (ARC) Program Annual Report. Since its launch in 2022, CDER’s ARC Program has become a key resource for the rare disease community and a driver of innovation in rare disease drug treatments. Having built a strong foundation, the ARC Program is enthusiastic about the upcoming year as it continues to strengthen collaboration to accomplish even more progress in rare disease drug development.

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Eplontersen approved to treat adults with rare inherited nerve disease

On 16th October, the MHRA announced the approval of eplontersen (Wainzua) to treat adults with polyneuropathy associated with hereditary transthyretin amyloidosis (ATTRv), which is a rare progressive condition that runs in families caused by a protein called transthyretin (TTR). Eplontersen works mainly by lowering the amount of TTR protein made by the liver and as a result, there is less TTR protein in the blood to form amyloid deposits.

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Elafibranor approved to treat adults with rare liver disease

On 16th October, the MHRA announced that Elafibranor (Iqirvo) has been approved to treat adult patients with a rare type of liver disease known as primary biliary cholangitis (PBC). Elafibranor helps to improve how the liver works by reducing the amount of bile acids the liver produces and reducing the build-up of bile. The approval is supported by evidence from a placebo-controlled main study involving 161 adults with PBC.

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Issue Number: WN00184

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