Tower Mains Weekly Newsletter
Industry Updates
FDA approves first device indicated for the preservation of donation-after-circulatory-death hearts
On 27th April, the US Food and Drug Administration (FDA) approved an expanded indication for the TransMedics Organ Care System (OCS) Heart System. This approval marks the first device indicated for the preservation of donation-after-circulatory-death (DCD) hearts in a warm, beating state. The OCS Heart System was previously indicated for the preservation of donation-after-brain-death (DBD) hearts considered unsuitable for procurement and transplantation at initial evaluation due to limitations of prolonged static cold storage. This expanded indication was granted a breakthrough designation for its intent to provide more effective treatment of a life-threatening or irreversibly debilitating disease or condition.
Ultomiris approved as treatment for adults with generalised myasthenia gravis
On 28th April, AstraZeneca announced the approval of Ultomiris for the treatment of adult patients with generalised myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody positive. The FDA action marks the first and only approval for a long-acting C5 complement inhibitor with early-onset and reliable efficacy.
ICH E8(R1) Introductory Training Presentation available on ICH website
On 28th April, the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) published a Step 4 Introductory Training Presentation to its website, which has been developed by the E8(R1) Expert Working Group.
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MHRA Compliance Monitor overview and application process
On 29th April, the Medicines and Healthcare products Regulatory Agency (MHRA) published guidance on its Compliance Monitor (CM) scheme, which is aimed at reducing potential shortages in the supply of safe medicines using risk-based supervision and monitoring. The process will also help the MHRA in concentrating resources on the delivery of the routine risk-based inspection programme, thus further ensuring patient safety.
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FDA guidance on how to submit electronic investigational new drug application safety reports
On 29th April, the FDA issued a Technical Conformance Guide providing specifications, recommendations, and general considerations on how to submit electronic investigational new drug application (IND) safety reports to the Center for Drug Evaluation and Research (CDER) or the Center for Biologics Evaluation and Research (CBER).
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FDA guidance on how to submit electronic investigational new drug application safety reports
On 29th April, the FDA published a technical specifications document to aid interested parties in electronically submitting individual case safety reports (ICSRs) to the CDER and CBER. The document describes the FDA’s technical approach for submitting ICSRs, for incorporating its regionally controlled terminology, and for adding FDA Adverse Event Reporting System (FAERS) regional data elements that are not addressed in the ICH E2B (R3) Implementation Guideline (IG).
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FDA guidance on use of circulating tumour deoxyribonucleic acid for early-stage drug development
On 2nd May, the FDA issued guidance to help sponsors planning to use circulating cell-free plasma-derived tumour deoxyribonucleic acid (ctDNA) as a biomarker in cancer clinical trials conducted under an investigational new drug application (IND) and/or to support marketing approval of drugs and biological products for treating solid tumour malignancies in the early-stage setting.
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ICH releases the Model-Informed Drug Development Discussion Group roadmap
On 4th May, the ICH released the Model-Informed Drug Development (MIDD) Discussion Group’s roadmap as an output of its discussions and considerations concerning future MIDD-related guidelines.
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Issue Number: WN00059
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