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Biotechnology
Emrelis (telisotuzumab vedotin-tllv) has been granted accelerated approval by the US Food and Drug Administration (FDA), said the drug’s developer, AbbVie. 15 May 2025
Biotechnology
Japanese pharma major Takeda announced that the US Food and Drug Administration (FDA) has accepted for priority review the supplemental Biologics License Application (sBLA) for Vonvendi [von Willebrand factor (Recombinant)] for investigational routine prophylactic use in adults (age 18 years and older) with von Willebrand disease (VWD), including those with Type 1 and 2 disease, and on-demand and perioperative use in children with VWD. 15 May 2025
Pharmaceutical
The US Food and Drug Administration approved Welireg (belzutifan), from US pharma giant Merck & Co, for adult and pediatric patients 12 years and older with locally advanced, unresectable, or metastatic pheochromocytoma or paraganglioma (PPGL). 15 May 2025
Biotechnology
Japanese drugmaker Chugai Pharmaceutical has obtained regulatory approval from the domestic regulator for Elevidys Intravenous Infusion (delandistrogene moxeparvovec) as a regenerative medicine product for the treatment of Duchenne muscular dystrophy (DMD) under the conditional and time-limited approval pathway. 15 May 2025
Biotechnology
UK-based Kaerus Bioscience, a biopharma created by European investment firm Medicxi for the development of therapeutics for rare genetic syndromes of neurodevelopment, has announced that its lead candidate KER-0193 has been granted both Orphan Drug Designation and Rare Pediatric Drug Designations for the treatment of Fragile X syndrome (FXS) by the US Food and Drugs Administration (FDA). 14 May 2025
Pharmaceutical
US pharma stocks regained ground Monday after Donald Trump signed an executive order aiming to slash prescription drug prices, a move that initially rattled investors but left many analysts unconvinced about its impact. 13 May 2025
Pharmaceutical
The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has approved Adzynma (apadamtase alfa/cinaxadamtase alfa; rADAMTS13), the first UK treatment to treat congenital thrombotic thrombocytopenic purpura (CTTP) in patients of all ages. 13 May 2025
Biotechnology
American health officials have recommended temporarily halting the use of Ixchiq (chikungunya vaccine) in people over 60 years old while investigations into reported serious adverse events continue. 12 May 2025
Pharmaceutical
UK pharma major AstraZeneca has suffered a setback in its legal fight to block the US government's efforts to lower prescription drug prices, after a federal appeals court dismissed its challenge to Medicare’s negotiation powers. 9 May 2025
Biotechnology
Belgium’s largest drugmaker UCB today revealed that it has received approval from Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) for at-home self-administration with infusion pump or a new manual push syringe method for Rystiggo (rozanolixizumab). 9 May 2025
Biotechnology
The US Food and Drug Administration (FDA) has approved Avmapki Fakzynja Co-pack (avutometinib capsules; defactinib tablets) for the treatment of adult patients with KRAS-mutated recurrent low-grade serous ovarian cancer (LGSOC) who received prior systemic therapy, Boston, said USA-based. Verastem Oncology, whose shares rose 3.7% to $6.94 on the news. 9 May 2025
Biotechnology
The European Commission (EC) has approved a Marketing Authorization (MA) for Tremfya (guselkumab) to treat adults with moderately to severely active Crohn’s disease (CD) who have had an inadequate response, lost response, or were intolerant to either conventional therapy or a biologic treatment. 8 May 2025
Pharmaceutical
Following a European Union-wide review of available data on finasteride and dutasteride medicines, the European Medicines Agency’s (EMA) safety committee (PRAC) has confirmed suicidal ideation (suicidal thoughts) as a side effect of finasteride 1mg and 5mg tablets but concluded that the benefits of finasteride and dutasteride medicines continue to outweigh their risks for all approved uses. 8 May 2025
Biotechnology
Sino-American biotech BeiGene today announced that the European Commission has approved Tevimbra (tislelizumab), in combination with etoposide and platinum chemotherapy, as a first-line treatment for adult patients with extensive-stage small cell lung cancer (ES-SCLC). 8 May 2025
Biotechnology
The American medicines regulator will convene its vaccine advisory committee on May 22 to weigh in on which strains should be used for the next round of COVID-19 booster shots, amid heightened interest in the new administration’s stance toward vaccines. 8 May 2025
Biotechnology
The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) yesterday approved Tepezza (teprotumumab (Tepezza). 8 May 2025
Biotechnology
California-based Atara Biotherapeutics revealed that the US Food and Drug Administration (FDA) has lifted the clinical hold on its active Investigational New Drug (IND) applications for the Ebvallo (tabelecleucel) program, sending the firm’s share up 5.8% to $7.67. 7 May 2025
Pharmaceutical
The US Food and Drug Administration (FDA) announced its intent to expand the use of unannounced inspections at foreign manufacturing facilities that produce foods, essential medicines, and other medical products intended for American consumers and patients. 7 May 2025
Biotechnology
US ophthalmics-focused biotech Opus Genetics today revealed that the US Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to OPGx-LCA5, its investigational gene therapy for the treatment of Leber congenital amaurosis (LCA) due to genetic variations in the LCA5 gene. 6 May 2025
Pharmaceutical
UK pharma major AstraZeneca today revealed that its Calquence (acalabrutinib) in combination with bendamustine and rituximab has been approved in the European Union (EU) for the treatment of adult patients with previously untreated mantle cell lymphoma (MCL) who are not eligible for autologous stem cell transplant. 6 May 2025
Biotechnology
The US Food and Drug Administration (FDA) announced the approval of 127 innovator and biosimilar drugs in 2024, a notable decrease from the 149 approvals recorded in 2023. 17 July 2025
Biotechnology
Swiss pharma giant Roche has won approval from the UK’s Medicines and Healthcare Regulatory Agency (MHRA) for its combination treatment of Columvi (glofitamab) alongside gemcitabine and oxaliplatin (GemOx) for adults with relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL) not otherwise specified who are ineligible for autologous stem cell transplant. 17 July 2025
Pharmaceutical
Artificial intelligence has edged past the pilot phase in clinical development. Once a niche add-on, AI is now helping biopharma companies design, recruit, and run trials faster, cheaper, and—crucially—more inclusively. And regulators are starting to pay serious attention. 17 July 2025
Biotechnology
UK pharma major GSK has gained US regulatory approval for a new prefilled syringe presentation of its shingles vaccine, Shingrix (zoster vaccine), a move aimed at streamlining administration and boosting immunization coverage. 17 July 2025
Biotechnology
UK pharma major GSK is seeking US approval for Blenrep (belantamab mafodotin) in two multiple myeloma indications. 16 July 2025
Biotechnology
The Division of Hepatology and Nutrition of the US Food and Drug Administration (FDA) has lifted the partial clinical hold on the completed PORTOLA Phase IIa clinical trial evaluating zetomipzomib, a first-in-class selective immunoproteasome inhibitor, in patients with autoimmune hepatitis (AIH), California, USA-based drug developer Kezar Life Sciences announced yesterday. 16 July 2025
Pharmaceutical
Alnylam has secured expanded UK regulatory approval for Amvuttra (vutrisiran), broadening its indication to include treatment of transthyretin amyloid cardiomyopathy (ATTR-CM) in adults. 16 July 2025
Pharmaceutical
The UK government’s new life sciences plan has been met with skepticism by the pharmaceutical industry, which says the strategy will fall flat unless there is a serious shift in the way new medicines are funded and valued. 16 July 2025
Biosimilars
The US Food and Drug Administration approved, the first rapid-acting interchangeable biosimilar product, Kirsty (insulin aspart-xjhz, as a 3 mL single-patient-use prefilled pen and 10 mL multiple-dose vial. Kirsty is biosimilar to and interchangeable with Novolog (insulin aspart), from Denmark’s Novo Nordisk. 16 July 2025
Pharmaceutical
China-based Jiangsu Hengrui Pharma and US start-up Kailera Therapeutics, which is advancing a broad pipeline of next-generation therapies for the treatment of obesity, today announced positive top-line data from Hengrui’s Phase III clinical trial (HRS9531-301) of once-weekly subcutaneous injection of HRS9531, a novel dual GLP-1/GIP receptor agonist, in individuals living with obesity or overweight in China. 15 July 2025
Pharmaceutical
The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has today approved Ekterly (sebetralstat) 0for the treatment of hereditary angioedema (HAE) attacks in adults and adolescents aged 12 years and older. 15 July 2025
Biotechnology
A quiet shift with potentially big implications for cancer drug access is underway in the UK. In June, the country’s health technology assessor, NICE, recommended GSK’s Blenrep (belantamab mafodotin) in multiple myeloma. 14 July 2025
Pharmaceutical
Germany’s Bayer has announced that the US Food and Drug Administration (FDA) has approved Kerendia (finerenone) to treat patients with heart failure (HF) with left ventricular ejection fraction (LVEF) ≥40%, following the agency’s Priority Review of its supplemental New Drug Application (sNDA). 14 July 2025
Pharmaceutical
The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has authorized the use of Bayer's Lynkuet (elinzanetant) for the treatment of moderate to severe vasomotor symptoms (VMS) associated with menopause. 14 July 2025
Generics
Even as the US Food and Drug Administration (FDA) has unveiled a strategic expansion of its oversight mechanisms for foreign manufacturing facilities supplying the American market, aiming to bolster product safety and ensure consistent quality for US consumers, the intensified oversight presents significant long-term benefits for the Indian pharmaceutical sector as a whole, reports The Pharma Letter’s India correspondent. 14 July 2025
Biosimilars
South Korea’s Samsung Bioepis today published a whitepaper titled ‘Solving the Biosimilar Void in Europe’ that proposes policy recommendations to fully recognize the value of biosimilars and ensure a more sustainable market in Europe. 14 July 2025
Pharmaceutical
US biotech Crinetics Pharmaceuticals has released new data on its investigational oral therapy Palsonify (paltusotine), supporting its potential as a convenient alternative to monthly injections for patients with acromegaly. 14 July 2025
Biotechnology
Ultragenyx Pharmaceutical has been dealt a regulatory setback, as the American medicines regulator has declined to approve its gene therapy UX111 (formerly ABO-102) for Sanfilippo syndrome type A. The decision, delivered in a complete response letter, will delay any potential approval until at least 2026. 14 July 2025
Biotechnology
US biotech Capricor Therapeutics today revealed receipt of a Complete Response Letter (CRL) from the US Food and Drug Administration (FDA) regarding its Biologics License Application (BLA) for deramiocel, the company’s lead cell therapy candidate for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy (DMD). 11 July 2025
Pharmaceutical
At its July meeting, the European Medicines Agency’s pharmacovigilance committee (PRAC) completed its review of French specialty vaccine maker Valneva Ixchiq (chikungunya virus vaccine, a live attenuated chikungunya vaccine, following reports of serious side effects. 11 July 2025
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