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What’s in Today’s Brief? (March 28th Preview)
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Regulatory approvals – gene therapy for ultra-rare immune deficiency
The FDA granted accelerated approval to Rocket Pharmaceuticals’ gene therapy Kresladi (marnetegragene autotemcel) for severe leukocyte adhesion deficiency-I (LAD-I), the first gene therapy option for the ultrarare immune disorder. The decision clears Kresladi for pediatric patients with severe LAD-I due to biallelic variants in ITGB2 who lack an HLA-matched sibling donor. The approval follows an earlier FDA rejection tied to manufacturing issues in 2024. With an April 2026 PDUFA date already in view for the original review path, Rocket’s resubmission and manufacturing remediation appear to have closed the regulatory gaps, enabling the accelerated pathway. For developers and investors watching rare-disease gene therapy, the key takeaway is that FDA’s manufacturing scrutiny—rather than efficacy alone—can still be decisive late in the process, even for first-in-category programs.
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Regulatory – EMA moves to withdraw mpox label
The EMA’s Committee for Medicinal Products for Human Use (CHMP) has recommended withdrawing the mpox indication for Siga Technologies’ tecovirimat, while keeping approval for smallpox, cowpox, and adverse reactions to vaccinia vaccines. The action follows CHMP’s review of the antiviral’s label after use in patients during recent mpox outbreaks. For commercialization and procurement planning, the decision narrows where tecovirimat can be promoted and reimbursed across European markets for mpox-specific use. It also underscores how quickly label positions can change once outbreak data, clinical outcomes, and public-health assumptions are revisited. Market participants will likely focus on whether Siga pursues additional evidence for mpox or shifts strategy toward its remaining approved indications.
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Drug approvals – Novo Nordisk’s first once-weekly basal insulin in the US
The FDA approved Novo Nordisk’s Awiqli (insulin icodec) injection 700 units/mL, making it the first and only once-weekly, long-acting basal insulin in the US. The approval gives Type 2 diabetes patients a longer-interval alternative to daily basal regimens. Novo’s path to the US finish line was shaped by regulatory review that began in 2023 and succeeded in other territories, setting up a US-specific milestone for the company’s basal insulin franchise. The label approval reinforces the commercial push for simpler dosing schedules in diabetes management. Clinicians and payers will now assess how once-weekly basal insulin fits into treatment algorithms relative to existing daily basal insulins and GLP-1–based combinations.
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M&A – Otsuka to acquire Transcend for a PTSD-focused neuroplasticity program
Otsuka is moving to buy Transcend Therapeutics for about $700 million upfront (roughly $1.2 billion total deal value), positioning the Japanese drugmaker to enter or expand psychiatric pipelines aimed at enhancing neuroplasticity. The transaction would be carried out through Otsuka’s American subsidiary and could include up to $525 million in additional milestones. Transcend’s lead program, TSND-201, is a methylone analog intended to rapidly and sustainably enhance neuroplasticity. In parallel with the deal, the company has pointed to a mid-stage PTSD dataset published in JAMA Psychiatry and is now running a Phase 3 trial in the US. The acquisition reflects broader pharma interest in psychiatric approaches that try to deliver faster functional rewiring—while avoiding some mechanistic issues associated with earlier MDMA-based efforts in PTSD.
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M&A – Novartis pays up to $2B for allergy biotech Excellergy
Novartis agreed to acquire Excellergy in a deal valued at up to $2 billion, bringing a next-generation anti-IgE program into the Swiss pharma’s allergy portfolio. Excellergy’s lead, Exl-111, is an effector cell response inhibitor (ECRI) that aims to suppress IgE signaling while avoiding triggering activation of mast cells and basophils. The deal follows Excellergy’s launch just months after a $70 million Series A, and the lead asset has entered Phase I clinical testing. Novartis framed Exl-111 as a potential Xolair successor strategy as competition intensifies, and emphasized faster and deeper IgE pathway suppression based on preclinical work and early pharmacokinetic data. For the broader immunology deal pipeline, the transaction is a reminder that platform-driven immunology startups can still attract large-ticket acquirers even at early clinical stages when differentiation and mechanism are clear.
...and 5 more selected Biotech stories in today’s full edition — or archive.
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