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Biotechnology
PTC Therapeutics is to sell its rare pediatric disease priority review voucher (PRV) for $150 million. 28 November 2024
Biotechnology
The UK subsidiary of Dutch drugmaker argenx confirmed today that its investigational therapy, subcutaneous efgartigimod alfa has been granted Promising Innovative Medicine (PIM) designation by the UK Medicines and Healthcare products Regulatory Agency (MHRA) for the treatment of adults with chronic inflammatory demyelinating polyneuropathy (CIDP). 28 November 2024
Pharmaceutical
The first iteration of the European Shortages Monitoring Platform (ESMP) has gone live. 28 November 2024
Pharmaceutical
The US Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) for the New Drug Application (NDA) for govorestat, a novel, central nervous system (CNS)-penetrant aldose reductase inhibitor (ARI), for the treatment of classic galactosemia, under development by Applied Therapeutics. 28 November 2024
Biotechnology
The US Food and Drug Administration has received new reports of hematologic malignancies, including life-threatening cases of myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML), following treatment of early, active cerebral adrenoleukodystrophy (CALD) patients with Skysona (elivaldogene autotemcel). 28 November 2024
Generics
The US Food and Drug Administration (FDA) yesterday announced its Fiscal Year 2025 Generic Drug User Fee Amendments (GDUFA). 28 November 2024
Pharmaceutical
UK-based drugmaker Advanz Pharma has announced that following the grant of a temporary suspension, as announced on last month, the General Court of the European Union (General Court) has decided not to further extend the suspension of the European Commission (EC) decision to revoke the conditional marketing authorization (CMA) for Ocaliva (obeticholic acid) in Europe. 28 November 2024
Biotechnology
The European medicines regulator has approved a combo therapy starring PD-1 inhibitor Tevimbra (tislelizumab), from cancer firm BeOne Medicines, formerly BeiGene. 27 November 2024
Biotechnology
After trading closed yesterday, Exelixis revealed that the US Food and Drug Administration (FDA) has notified the company that the supplemental New Drug Application (sNDA) for cabozantinib (Cabometyx) for the treatment of adults with previously treated advanced pancreatic neuroendocrine tumors (pNET) and advanced extra-pancreatic NET (epNET) will be discussed at an Oncologic Drugs Advisory Committee (ODAC) meeting in March 2025. 27 November 2024
Pharmaceutical
The European Commission (EC) has approved a single-vial, fully liquid presentation of Menveo (meningococcal Group A, C, W-135 and Y conjugate vaccine, MenACWY vaccine) to help protect against invasive meningococcal disease (IMD) caused by bacterial serogroups A, C, W and Y, UK pharma major GSK announced today. 27 November 2024
Pharmaceutical
Novartis announced today that the European Commission (EC) has approved Kisqali (ribociclib) in combination with an aromatase inhibitor (AI) for the adjuvant treatment of patients with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative early breast cancer (EBC) at high risk of recurrence. 27 November 2024
Biotechnology
Chengdu-based Kelun-Biotech has received marketing authorization in China for sacituzumab tirumotecan (sac-TMT), a Trop2-directed antibody-drug conjugate (ADC). 27 November 2024
Pharmaceutical
The Biden administration has proposed a new rule that could significantly expand Medicare and Medicaid coverage for obesity medications, potentially benefiting millions of Americans. 27 November 2024
Pharmaceutical
The US Food and Drug Administration (FDA) has extended the review period for Soleno Therapeutics’ the New Drug Application (NDA) for DCCR (diazoxide choline) extended-release tablets 27 November 2024
Pharmaceutical
Japanese drugmaker Otsuka has announced the submission of a New Drug Application (NDA) to the Japanese Ministry of Health, Labor and Welfare (MHLW) for the manufacture and sale in Japan of bempedoic acid - trade name Nexletol in Japan - for the treatment of hypercholesterolemia and familial hypercholesterolemia. 27 November 2024
Pharmaceutical
Berlin, Germany-based Eckert & Ziegler Radiopharma has received approval of its proprietary non-carrier added Lutetium-177 chloride, Theralugand, by the European Commission (EC). 27 November 2024
Biotechnology
The US Food and Drug Administration (FDA) has accepted for review a Biologics License Application (BLA) for belantamab mafodotin in combinations with bortezomib plus dexamethasone (BorDex [BVd]) and pomalidomide plus dexamethasone (PomDex [BPd]) for multiple myeloma patients who have received at least one prior line of therapy. 25 November 2024
Pharmaceutical
The US Food and Drug Administration (FDA) has approved Imkeldi (imatinib) oral solution, the first oral liquid form of imatinib to treat certain forms of leukemia and other cancers, from privately-held US-Ireland-based pharmaceutical company Shorla Oncology. 25 November 2024
Biotechnology
StemCyte, a subsidiary of StemCyte International, has secured approval from the US Food and Drug Administration for Regenecyte, a cord blood-derived stem cell therapy. 25 November 2024
Pharmaceutical
In what could be construed to be India's quest for pharmaceutical supremacy, a clarion call has been made for the country to set its indigenous standards. 25 November 2024
Pharmaceutical
After a first early benefit assessment in April 2012, the German Institute for Quality and Efficiency in Health Care (IQWiG) has now re-examined whether Nulojix (belatacept) offers an added benefit over the appropriate comparator therapy. 21 October 2015
Biotechnology
The US Food and Drug Administration yesterday approved Coagadex, coagulation Factor X (human), for hereditary Factor X (10) deficiency. 21 October 2015
Pharmaceutical
The National Institute for Health and Care Excellence (NICE) has today published draft guidance recommending US pharma giant Merck & Co’s Ezetrol (ezetimibe) as an option for some adults with primary (heterozygous-familial and non-familial) hypercholesterolemia. 21 October 2015
Biotechnology
The German Institute for Quality and Efficiency in Health Care (IQWiG) has examined in a dossier assessment whether US pharma major Bristol-Myers Squibb’s Opdivo (nivolumab) offers an added benefit over the appropriate comparator therapy for advanced melanoma. 20 October 2015
Pharmaceutical
The Food and Drug Administration has granted Breakthrough Therapy designation for US pharma giant Pfizer’s once failed inotuzumab ozogamicin for the treatment of acute lymphoblastic leukemia (ALL). 20 October 2015
Pharmaceutical
LEO Pharma said today that its Enstilar (calcipotriene/betamethasone dipropionate) foam has been approved by the US Food and Drug Administration for the topical treatment of plaque psoriasis in adults 18 years of age and older. 19 October 2015
Pharmaceutical
The Netherland’s Medicines Evaluation Board (MEB) has completely revised the policy document on the nomenclature of medicinal products for humans. 19 October 2015
Pharmaceutical
Shire revealed on Friday that the US Food and Drug Administration has requested an additional clinical study as part of a complete response letter (CRL) to the company’s new drug application for Lifitegrast for the signs and symptoms of dry eye disease in adults. 17 October 2015
Pharmaceutical
The US Food and Drug Administration today granted accelerated approval to Praxbind (idarucizumab) for use in patients who are taking the anticoagulant Pradaxa (dabigatran) during emergency situations when there is a need to reverse Pradaxa’s blood-thinning effects. 17 October 2015
Biotechnology
PTC Therapeutics has announced disappointing results from the Phase III, double-blind, placebo-controlled, 48-week ACT DMD trial of Translarna (ataluren), an oral, first-in-class, protein restoration therapy for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). 16 October 2015
Biotechnology
US biotech firm Zafgen has received verbal notice from the US Food and Drug Administration that beloranib has been placed on partial clinical hold. 16 October 2015
Pharmaceutical
The US Food and Drug Administration has issued a complete response letter (CRL) regarding Anglo-Swedish pharma company AstraZeneca’s New Drug Application for the investigational fixed-dose combination of saxagliptin and dapagliflozin for the treatment of adult patients with type 2 diabetes. 16 October 2015
Pharmaceutical
In final draft guidance issued today, the National Institute for Health and Care Excellence (NICE), the medicines watchdog for England and Wales, has recommended Danish CNS specialist Lundbeck’s Brintellix (vortioxetine) for some adults with major depressive disorder 16 October 2015
Biotechnology
Swedish Orphan Biovitrum has received approval from Swissmedic, the Swiss Agency for Therapeutic Products, for Xiapex (collagenase clostridium histolyticum) for the treatment of adult men with Peyronie's disease. 16 October 2015
Biotechnology
In draft guidance produced by its Highly Specialised Technologies program, England and Wales medicines watchdog the National Institute for Health and Care Excellence (NICE) has today asked for further clarification from US biotech firm PCT Therapeutics on the size of the benefit its drug Translarna (ataluren) provides in the treatment of a type of Duchenne muscular dystrophy (DMD). 16 October 2015
Biotechnology
Following preliminary positive recommendations announced in July, The National Institute for Health and Care Excellence (NICE), the medicines watchdog for England and Wales, is now set to recommend three new treatments for some adults with hepatitis C, the agency announced today. 16 October 2015
Pharmaceutical
Baxalta says that medicines regulator Health Canada has approved its Obizur (antihemophilic Factor [recombinant], porcine sequence) for the treatment of bleeding episodes in patients with acquired hemophilia A. 15 October 2015
Pharmaceutical
Ampio Pharmaceutical announced it had met with the Division of the Transplant and Ophthalmology Products of the Food and Drug Administration to discuss the results of the OptimEyes clinical trial of Optina (ultra-low dose danazol) and to seek guidance on the next steps to approval. 14 October 2015
Pharmaceutical
The UK’s National Institute for Health and Care Excellence (NICE) is introducing a new service to speed up the adoption of new medicines, devices and diagnostics by the UK’s National Health Service (NHS). 13 October 2015
Pharmaceutical
The US Food and Drug Administration has advised Collegium Pharmaceuticals that it will not be able to complete its review of the New Drug Application for Xtampza ER (oxycodone) extended-release capsules by the Prescription Drug User Fee Act (PDUFA) action date of October 12, 2015. 13 October 2015
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