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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
Biotechnology
Shares in US biotech Ultragenyx Pharmaceutical and its UK-based partner Mereo BioPharma tumbled after the companies confirmed their pivotal trial in osteogenesis imperfecta (OI) would not be stopped early, defying investor hopes for a quicker path to approval. 11 July 2025
Biotechnology
Japan’s JCR Pharmaceuticals closed 15% higher after Wednesday’s trading, as it signed a new licensing deal. 9 July 2025
Biotechnology
Having appointed Renée Aguiar-Lucander as its chief executive in April and Maria Törnsén as chief operating officer a month later, Hansa Biopharma has announced the appointment of a chief medical officer (CMO). 8 July 2025
Pharmaceutical
Shares in Japanese drugmaker Otsuka Holdings closed nearly 2% lower Tuesday, after disappointing clinical trial results from one of its subsidiaries. 8 July 2025
Pharmaceutical
USA and UK-based KalVista Pharmaceuticals today announced that the US Food and Drug Administration (FDA) has approved Ekterly (sebetralstat), a novel plasma kallikrein inhibitor, for the treatment of acute attacks of hereditary angioedema (HAE) in adult and pediatric patients aged 12 years and older. 7 July 2025
Biotechnology
US biopharma Apellis Pharmaceuticals announced a capped royalty purchase agreement with Swedish Orphan Biovitrum where Apellis will receive up to $300 million in exchange for 90% of Apellis’ future ex-US royalties for Aspaveli (systemic pegcetacoplan). 3 July 2025
Biotechnology
US biopharma Syntis Bio, which is developing oral therapies for obesity, diabetes and rare diseases, has announced the close of a $33 million oversubscribed Series A financing. 2 July 2025
Pharmaceutical
Alnylam Pharmaceuticals has won European Commission approval for Amvuttra (vutrisiran) to treat transthyretin amyloidosis with cardiomyopathy (ATTR-CM), positioning the drug as a likely challenger to entrenched therapies in a market expected to see rapid evolution. 2 July 2025
Pharmaceutical
Vertex Pharmaceuticals today announced that the European Commission has granted approval for Alyftrek (deutivacaftor/tezacaftor/vanzacaftor) for the treatment of people with cystic fibrosis (CF) ages six years and older who have at least one non-class I mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. 1 July 2025
Pharmaceutical
Swedish biotech Camurus has secured EU marketing authorization for its once-monthly acromegaly therapy, Oczyesa (octreotide), marking a significant step forward in treatment convenience for patients living with the rare endocrine disorder. 1 July 2025
Biotechnology
Systemic sclerosis is an autoimmune connective tissue disease that causes debilitating fibrosis of the skin and internal organs. It impacts nearly 50,000 people in the USA alone1 and between 7.2 and 33.9 per 100,000 individuals across Europe.2 1 July 2025
Biotechnology
North Carolina, USA-based biotech BioCryst Pharmaceuticals on Friday announced it has signed a definitive agreement to sell its European Orladeyo (berotralstat) business to Italy’s Neopharmed Gentili for up to $264 million. 28 June 2025
article
Swedish Orphan Biovitrum, the Nordic biotech also known as Sobi on Frida,y announced that the US Food and Drug Administration (FDA) approved Gamifant (emapalumab-lzsg) for a new indication, edging the company’s shares up 2.4% to 284.60 kronor. 28 June 2025
Biotechnology
US biotech major Biogen today announced new data that reinforce the clinical impact of nusinersen across a broad spectrum of individuals affected by spinal muscular atrophy (SMA). 27 June 2025
Pharmaceutical
Belgian pharma UCB today announced that the Phase III study investigating the safety and efficacy of adjunctive fenfluramine in CDKL5 deficiency disorder (CDD) met its primary and key secondary endpoints. 27 June 2025
Biotechnology
Australian biotech CSL Limited Europe-based subsidiary CSL Behring announced a milestone in the early benefit assessment of garadacimab by Germany’s Institute for Quality and Efficiency in Health Care (IQWiG). 26 June 2025
Biotechnology
US biotech PTC Therapeutics’ (Nasdaq: PTCT) Sephience (sepiapterin) has been granted marketing authorization by the European Commission for the treatment of children and adults living with phenylketonuria (PKU). The authorization includes a broad label inclusive of all ages and disease severities. 25 June 2025
Biotechnology
European biotech argenx has secured European Commission approval for Vyvgart (efgartigimod alfa) in chronic inflammatory demyelinating polyneuropathy (CIDP), marking the first regulatory green light for a novel mechanism in this condition in over three decades. 23 June 2025
Pharmaceutical
US biopharma Vertex Pharmaceuticals and Japanese drugmaker Ono Pharmaceutical have announced an exclusive collaboration and license agreement for the development and commercialization of Vertex’s povetacicept in Japan and South Korea. 23 June 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
Biotechnology
Shares in US biotech Ultragenyx Pharmaceutical and its UK-based partner Mereo BioPharma tumbled after the companies confirmed their pivotal trial in osteogenesis imperfecta (OI) would not be stopped early, defying investor hopes for a quicker path to approval. 11 July 2025
Biotechnology
Japan’s JCR Pharmaceuticals closed 15% higher after Wednesday’s trading, as it signed a new licensing deal. 9 July 2025
Biotechnology
Having appointed Renée Aguiar-Lucander as its chief executive in April and Maria Törnsén as chief operating officer a month later, Hansa Biopharma has announced the appointment of a chief medical officer (CMO). 8 July 2025
Pharmaceutical
Shares in Japanese drugmaker Otsuka Holdings closed nearly 2% lower Tuesday, after disappointing clinical trial results from one of its subsidiaries. 8 July 2025
Pharmaceutical
USA and UK-based KalVista Pharmaceuticals today announced that the US Food and Drug Administration (FDA) has approved Ekterly (sebetralstat), a novel plasma kallikrein inhibitor, for the treatment of acute attacks of hereditary angioedema (HAE) in adult and pediatric patients aged 12 years and older. 7 July 2025
Biotechnology
US biopharma Apellis Pharmaceuticals announced a capped royalty purchase agreement with Swedish Orphan Biovitrum where Apellis will receive up to $300 million in exchange for 90% of Apellis’ future ex-US royalties for Aspaveli (systemic pegcetacoplan). 3 July 2025
Biotechnology
US biopharma Syntis Bio, which is developing oral therapies for obesity, diabetes and rare diseases, has announced the close of a $33 million oversubscribed Series A financing. 2 July 2025
Pharmaceutical
Alnylam Pharmaceuticals has won European Commission approval for Amvuttra (vutrisiran) to treat transthyretin amyloidosis with cardiomyopathy (ATTR-CM), positioning the drug as a likely challenger to entrenched therapies in a market expected to see rapid evolution. 2 July 2025
Pharmaceutical
Vertex Pharmaceuticals today announced that the European Commission has granted approval for Alyftrek (deutivacaftor/tezacaftor/vanzacaftor) for the treatment of people with cystic fibrosis (CF) ages six years and older who have at least one non-class I mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. 1 July 2025
Pharmaceutical
Swedish biotech Camurus has secured EU marketing authorization for its once-monthly acromegaly therapy, Oczyesa (octreotide), marking a significant step forward in treatment convenience for patients living with the rare endocrine disorder. 1 July 2025
Biotechnology
Systemic sclerosis is an autoimmune connective tissue disease that causes debilitating fibrosis of the skin and internal organs. It impacts nearly 50,000 people in the USA alone1 and between 7.2 and 33.9 per 100,000 individuals across Europe.2 1 July 2025
Biotechnology
North Carolina, USA-based biotech BioCryst Pharmaceuticals on Friday announced it has signed a definitive agreement to sell its European Orladeyo (berotralstat) business to Italy’s Neopharmed Gentili for up to $264 million. 28 June 2025
article
Swedish Orphan Biovitrum, the Nordic biotech also known as Sobi on Frida,y announced that the US Food and Drug Administration (FDA) approved Gamifant (emapalumab-lzsg) for a new indication, edging the company’s shares up 2.4% to 284.60 kronor. 28 June 2025
Biotechnology
US biotech major Biogen today announced new data that reinforce the clinical impact of nusinersen across a broad spectrum of individuals affected by spinal muscular atrophy (SMA). 27 June 2025
Pharmaceutical
Belgian pharma UCB today announced that the Phase III study investigating the safety and efficacy of adjunctive fenfluramine in CDKL5 deficiency disorder (CDD) met its primary and key secondary endpoints. 27 June 2025
Biotechnology
Australian biotech CSL Limited Europe-based subsidiary CSL Behring announced a milestone in the early benefit assessment of garadacimab by Germany’s Institute for Quality and Efficiency in Health Care (IQWiG). 26 June 2025
Biotechnology
US biotech PTC Therapeutics’ (Nasdaq: PTCT) Sephience (sepiapterin) has been granted marketing authorization by the European Commission for the treatment of children and adults living with phenylketonuria (PKU). The authorization includes a broad label inclusive of all ages and disease severities. 25 June 2025
Biotechnology
European biotech argenx has secured European Commission approval for Vyvgart (efgartigimod alfa) in chronic inflammatory demyelinating polyneuropathy (CIDP), marking the first regulatory green light for a novel mechanism in this condition in over three decades. 23 June 2025
Pharmaceutical
US biopharma Vertex Pharmaceuticals and Japanese drugmaker Ono Pharmaceutical have announced an exclusive collaboration and license agreement for the development and commercialization of Vertex’s povetacicept in Japan and South Korea. 23 June 2025
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