Latest Pharma and Biotech News
Pharmaceutical
2 April 2025 Freed from their former obligations to remain loyal to agency and state, a number of high-profile figures from the US Food and Drug Administration (FDA) have sounded the alarm about its direction of travel under Robert Francis Kennedy Jr (RFK Jr), the new Secretary of Health and Human Services.
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Pharmaceutical
The waiting time for Latin American patients with cancer, central nervous system disorders or rare diseases can take up to 4.75 years from the time a drug receives approval from global health authorities such as the US Food and Drug Administration (FDA) or the European Medicines Agency (EMA), until it is approved by their local authority and is available on the market, writes The Pharma Letter's local correspondent. 28 November 2024
Biotechnology
PTC Therapeutics is to sell its rare pediatric disease priority review voucher (PRV) for $150 million. 28 November 2024
Biotechnology
A report from industry analyst DelveInsight highlights progress in the myotonic dystrophy treatment landscape, with more than 20 companies developing over 22 innovative treatments. 28 November 2024
Pharmaceutical
A new indication for olverembatinib, developed by Suzhou-based Ascentage Pharma will be added to China’s National Reimbursement Drug List (NRDL) from next year. 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
Privately-held French drugmaker Servier has announced the inauguration of its first unit dedicated to the production of biological medicines from its R&D pipeline. 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
Biotechnology
San Diago, USA-based Acadia Pharmaceuticals yesterday announced it has entered into an exclusive worldwide license agreement with Denmark’s Saniona for the development and commercialization of SAN711, a first-in-class, highly selective GABAA-α3 positive allosteric modulator. 28 November 2024
Pharmaceutical
Munich, Germany headquartered contract development and manufacturing organization (CDMO) Adragos Pharma has acquired Baccinex, a Swiss-based expert in aseptic fill-finish manufacturing. 28 November 2024
Pharmaceutical
The board of Spanish plasma-based medicines producer Grifols has announced the termination of discussions with Brookfield Capital Partners (UK) regarding a potential acquisition of Grifols shares. 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
Biotechnology
Kronos Bio, a biotech with a presence on the east and west coasts of the USA, has unveiled a major restructuring plan. 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
Johnson & Johnson has made significant strides in multiple myeloma treatment, with breakthroughs in targeted therapies like CAR-T and bispecific antibodies. At the 2024 ASH meeting, J&J aims to showcase progress, with a goal of achieving a functional cure for multiple myeloma. 28 November 2024
Biotechnology
The European medicines regulator has approved a combo therapy starring PD-1 inhibitor Tevimbra (tislelizumab), from cancer firm 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
According to a report from industry analyst DelveInsight, the market for Down syndrome treatments in the USA is now valued at almost $250 million. 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
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Pharmaceutical
US pharma major Eli Lilly’s brand value has surged by 108% since entering the weight loss market in 2023, according to a new report from Brand Finance. 3 April 2025
Biotechnology
US biotech BioMarin Pharmaceutical yesterday revealed that the Phase III PEGASUS trial evaluating Palynziq (pegvaliase-pqpz) met its primary efficacy endpoint, demonstrating a statistically-significant lowering in blood phenylalanine( Phe) levels in adolescents aged 12-17 with phenylketonuria (PKU) compared to diet alone. 3 April 2025
Biotechnology
Sino-American biotech BeiGene today revealed it intends to discontinue its clinical development program for ociperlimab (BGB-A1217), an anti-TIGIT antibody, as a potential treatment for lung cancer. 3 April 2025
Biotechnology
Australian biotech major CSL Limited subsidiary CSL Behring today announced that it has successfully concluded negotiations in Germany with the GKV-Spitzenverband (National Association of Statutory Health Insurance Funds) resulting in national reimbursement of Hemgenix (etranacogene dezaparvovec). 3 April 2025
Pharmaceutical
Colorado, USA-based Edgewise Therapeutics, a muscle disease-focused biopharma company, closed 23% lower after Wednesday’s trading. 3 April 2025
Pharmaceutical
With two new lawsuits, Eli Lilly has escalated its legal campaign against the makers of unauthorized versions of its diabetes and weight-loss drugs, Mounjaro (tirzepatide) and Zepbound (tirzepatide), challenging inroads made by drug compounders in what has become a highly lucrative market. 3 April 2025
Generics
US generic manufacturer Padagis has received clearance to extend the shelf life of its over-the-counter naloxone nasal spray to 36 months, as the company looks to bolster value and accessibility in a crowded but critical public health market. 3 April 2025
Pharmaceutical
Pharmaceuticals will be exempt from the tariffs on imports to the USA that were announced by US President Donald Trump Wednesday, according to a White House fact sheet. 3 April 2025
Pharmaceutical
Novartis today announced the US Food and Drug Administration (FDA) has granted accelerated approval for Vanrafia (atrasentan), a potent and selective endothelin A (ETA) receptor antagonist, for the reduction of proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression. 3 April 2025
Biotechnology
US clinical-stage biotech Nurix Therapeutics announced that Sanofi has exclusively licensed an undisclosed Nurix program targeting a previously undruggable transcription factor for autoimmune diseases. 3 April 2025
Biotechnology
North Carolina, USA-based clinical-stage gene therapy company Atsena Therapeutics has announced the successful closing of an oversubscribed $150 million Series C financing. 3 April 2025
Biotechnology
Swiss clinical-stage biotech AC Immune’s shares closed up 5.9% at $1.90 yesterday, after it announced additional interim safety and positive immunogenicity data from the Phase II VacSYn clinical trial evaluating ACI-7104.056, its wholly owned anti-alpha-synuclein (a-syn) active immunotherapy candidate, for the treatment of patients with early Parkinson’s disease (PD). 3 April 2025
Biotechnology
The European Medicines Agency has temporarily suspended enrollment and dosing in a Phase II trial of Sarepta Therapeutics' gene therapy, Elevidys (delandistrogene moxeparvovec-rokl), for Duchenne muscular dystrophy (DMD). 3 April 2025
Pharmaceutical
CNS specialist Axsome Therapeutics has announced results from the PARADIGM Phase III proof-of-concept trial of solriamfetol in major depressive disorder (MDD) with and without severe excessive daytime sleepiness (EDS). 3 April 2025
Biotechnology
Swiss pharma major Roche said a higher-dose version of its top-selling multiple sclerosis drug Ocrevus (ocrelizumab) failed to deliver additional benefit in slowing disability progression, compared to the currently approved dose, in a Phase III trial. 3 April 2025
Biotechnology
US biotech Inhibrx Biosciences has announced a leadership shake-up following the departure of co-founder Brendan Eckelman, who is leaving to establish a new privately-held biotech company. 2 April 2025
Biotechnology
Swedish Orphan Biovitrum, the Nordic biotech also known as Sobi, has announced that the National Institute for Health and Care Excellence (NICE) has issued its final guidance in England and Wales recommending the use of Altuvoct (efanesoctocog alfa). 2 April 2025
Biotechnology
US biotech Artis BioSolutions has officially launched with the acquisition of Landmark Bio, a translational development and manufacturing group formed through an alliance of academic and industry stakeholders. 2 April 2025
Biotechnology
The UK’s health technology assessor the National Institute for Health and Care Excellence (NICE) has recommended brentuximab vedotin in combination with doxorubicin, vinblastine and dacarbazine (AVD) to treat adult patients with untreated stage 3 or 4 CD30+ Hodgkin lymphoma (HL), within final draft guidance (FDG). 2 April 2025
Generics
German tech conglomerate Siemens has signed an agreement with Miko Pharma to develop facilities for the local production of generic pharmaceuticals in Ghana. 2 April 2025
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