Pharma & Drug Safety

Cerliponase alfa for Neuronal Ceroid Lipofuscinosis Type 2 Guidance

NICE has published updated highly specialised technologies guidance (HST34) on cerliponase alfa (Brineura) for treating neuronal ceroid lipofuscinosis type 2 in children. This guidance replaces previous guidance and confirms that patients already receiving treatment can continue.

NICE Guidance: Pegzilarginase for Arginase-1 Deficiency

NICE has published guidance recommending pegzilarginase (Loargys) for treating arginase-1 deficiency in individuals aged 2 years and over. The guidance includes evidence-based recommendations and details a commercial access agreement for the treatment.

NICE Guidance: Durvalumab for Muscle-Invasive Bladder Cancer

NICE has published technology appraisal guidance (TA1138) recommending durvalumab with gemcitabine and cisplatin for the neoadjuvant and adjuvant treatment of muscle-invasive bladder cancer. The guidance includes information on commercial arrangements and reporting adverse events.

NICE Guidance: Epcoritamab for Follicular Lymphoma

NICE has published technology appraisal guidance (TA1139) recommending epcoritamab (Tepkinly) for treating relapsed or refractory follicular lymphoma in adults after two or more lines of systemic treatment. The guidance outlines evidence-based recommendations for healthcare professionals.

Canagliflozin Appraisal Terminated for Type 2 Diabetes in Youth

NICE has terminated the appraisal of canagliflozin (Invokana) for treating type 2 diabetes in individuals aged 10 to 17 years. The appraisal was terminated because the company failed to provide an evidence submission. NICE will review this decision if a submission is made.

Baloxavir marboxil appraisal terminated for children

NICE has terminated the appraisal of baloxavir marboxil for treating and preventing influenza in children aged 1 to 11 years. The decision was made because the company failed to provide an evidence submission for the technology appraisal.

NICE Guidance on Bevacizumab for Metastatic Colorectal Cancer (TA1136)

NICE has published updated technology appraisal guidance (TA1136) on the use of bevacizumab (originator and biosimilars) with fluoropyrimidine-based chemotherapy for metastatic colorectal cancer. This guidance replaces previous recommendations and provides evidence-based recommendations for healthcare professionals.

Dapagliflozin Tablets Monograph Revision Notice

The USP is issuing a notice of intent to revise the Dapagliflozin Tablets monograph, with a targeted official date of June 1, 2026. This revision is being made in response to comments received, aiming to incorporate feedback into the monograph.

Carbachol Monograph Revision - Assay Criteria

The United States Pharmacopeia (USP) is revising the Carbachol monograph to widen the acceptance criteria for Assay from NMT 99.0%-101.0% to NMT 98.0%-102.0%. This change aims to accommodate FDA-approved products with wider specifications and is targeted for publication on May 29, 2026, with an official date of June 1, 2026.

Adapalene Monograph Revision Proposed for Solubility Issue

The USP Small Molecules 3 Expert Committee intends to revise the Adapalene monograph to address a potential solubility issue in the Organic Impurities test. The proposed changes include allowing a larger volume of tetrahydrofuran for the standard stock solution, introducing a two-table format for retention times, and deleting the Limit of Triethylamine test.