Biopharma

Orphan designation: Clofutriben Treatment of Cushing's syndrome of endogenous origin, 16/01/2025 Positive

Orphan designation: Clofutriben Treatment of Cushing&#0...

Feb 20, 2025 0

Orphan designation: Clofutriben Treatment of Cushing's syndrome of endogenous or...

FDA hits pair of Indian API makers with warning letters, import alerts

FDA hits pair of Indian API makers with warning letters...

Feb 20, 2025 0

Jagsonpal Pharmaceuticals and Tyche Industries, both makers of APIs based in Ind...

AstraZeneca pays $160M for FibroGen's China unit, regional rights to roxadustat

AstraZeneca pays $160M for FibroGen's China unit, regio...

Feb 20, 2025 0

AstraZeneca is paying $160 million for FibroGen’s Chinese business, giving the U...

Orphan designation: zopapogene imadenovec Treatment of recurrent respiratory papillomatosis, 12/01/2024 Positive

Orphan designation: zopapogene imadenovec Treatment of ...

Feb 20, 2025 0

Orphan designation: zopapogene imadenovec Treatment of recurrent respiratory pap...

Orphan designation: N-(2-Methoxyethyl)-6-methyl-N-[(3-methyl-2-thienyl)methyl]-2-oxo-1,2-dihydropyridine-4-carboxamide Treatment of glycogen storage disease type IV, 16/01/2025 Positive

Orphan designation: N-(2-Methoxyethyl)-6-methyl-N-[(3-m...

Feb 20, 2025 0

Orphan designation: N-(2-Methoxyethyl)-6-methyl-N-[(3-methyl-2-thienyl)methyl]-2...

Human medicines European public assessment report (EPAR): Tuznue, trastuzumab, Date of authorisation: 19/09/2024, Revision: 1, Status: Authorised

Human medicines European public assessment report (EPAR...

Feb 20, 2025 0

Human medicines European public assessment report (EPAR): Tuznue, trastuzumab, D...

Orphan designation: Adeno-associated viral vector serotype 9 containing the human N-acetylglucosaminidase-alpha gene Treatment of mucopolysaccharidosis type IIIB (Sanfilippo B syndrome), 24/01/2013 Withdrawn

Orphan designation: Adeno-associated viral vector serot...

Feb 20, 2025 0

Orphan designation: Adeno-associated viral vector serotype 9 containing the huma...

Orphan designation: Autologous CD34+ cells transduced with a lentiviral vector containing the human RAG1 gene Treatment of recombination-activating gene 1 deficient severe combined immunodeficiency, 26/03/2014 Positive

Orphan designation: Autologous CD34+ cells transduced w...

Feb 20, 2025 0

Orphan designation: Autologous CD34+ cells transduced with a lentiviral vector c...

Orphan designation: Autologous CD34+ cells transfected with lentiviral vector containing the Wiskott-Aldrich syndrome protein gene (etuvetidigene autotemcel) Treatment of Wiskott-Aldrich syndrome, 07/10/2013 Withdrawn

Orphan designation: Autologous CD34+ cells transfected ...

Feb 20, 2025 0

Orphan designation: Autologous CD34+ cells transfected with lentiviral vector co...

Orphan designation: amitriptyline Treatment of erythromelalgia, 21/06/2022 Withdrawn

Orphan designation: amitriptyline Treatment of erythrom...

Feb 20, 2025 0

Orphan designation: amitriptyline Treatment of erythromelalgia, 21/06/2022 Withd...

Orphan designation: Adeno-associated viral vector serotype 9 containing the human iduronate-2-sulfatase gene Treatment of mucopolysaccharidosis type II (Hunter's syndrome), 10/08/2015 Withdrawn

Orphan designation: Adeno-associated viral vector serot...

Feb 20, 2025 0

Orphan designation: Adeno-associated viral vector serotype 9 containing the huma...

Orphan designation: Adeno-associated virus serotype 5 vector encoding C1-esterase inhibitor Treatment of hereditary angioedema, 10/11/2022 Withdrawn

Orphan designation: Adeno-associated virus serotype 5 v...

Feb 20, 2025 0

Orphan designation: Adeno-associated virus serotype 5 vector encoding C1-esteras...

Orphan designation: xevinapant Treatment of ovarian cancer, 11/11/2015 Withdrawn

Orphan designation: xevinapant Treatment of ovarian can...

Feb 20, 2025 0

Orphan designation: xevinapant Treatment of ovarian cancer, 11/11/2015 Withdrawn

Orphan designation: volixibat potassium Treatment of primary biliary cholangitis, 16/01/2025 Positive

Orphan designation: volixibat potassium Treatment of pr...

Feb 20, 2025 0

Orphan designation: volixibat potassium Treatment of primary biliary cholangitis...

Human medicines European public assessment report (EPAR): Imfinzi, durvalumab, Date of authorisation: 21/09/2018, Revision: 24, Status: Authorised

Human medicines European public assessment report (EPAR...

Feb 20, 2025 0

Human medicines European public assessment report (EPAR): Imfinzi, durvalumab, D...

Orphan designation: Allogenic umbilical cord-derived osteoblast cells Treatment of non-traumatic osteonecrosis, 16/01/2025 Positive

Orphan designation: Allogenic umbilical cord-derived os...

Feb 20, 2025 0

Orphan designation: Allogenic umbilical cord-derived osteoblast cells Treatment ...

Orphan designation: Elraglusib Treatment of soft tissue sarcoma, 16/01/2025 Positive

Orphan designation: Elraglusib Treatment of soft tissue...

Feb 20, 2025 0

Orphan designation: Elraglusib Treatment of soft tissue sarcoma, 16/01/2025 Posi...

Genentech, Novartis join forces to raise awareness of food allergies’ impact on families

Genentech, Novartis join forces to raise awareness of f...

Feb 20, 2025 0

Genentech and Novartis have teamed up with FARE to enhance the safety of childre...

Orphan designation: [4-(Methyl-1H-pyrazol-4-yl)-benzyl]-(6[7-(3-pyrrolidin-1-yl-propoxy)-imidazo[1,2-a]pyridin-3-yl]-pyrimidin-4-yl]-amine Treatment of gastrointestinal stromal tumours, 16/01/2025 Positive

Orphan designation: [4-(Methyl-1H-pyrazol-4-yl)-benzyl]...

Feb 20, 2025 0

Orphan designation: [4-(Methyl-1H-pyrazol-4-yl)-benzyl]-(6[7-(3-pyrrolidin-1-yl-...

AstraZeneca pays $160M for Fibrogen's China unit, regional rights to roxadustat

AstraZeneca pays $160M for Fibrogen's China unit, regio...

Feb 20, 2025 0

AstraZeneca is paying $160 million for Fibrogen’s Chinese business, giving the U...

DARWIN EU data partners onboarded in phases I, II and III

Feb 20, 2025 0

DARWIN EU data partners onboarded in phases I, II and III

Orphan designation: Elraglusib Treatment of pancreatic cancer, 16/01/2025 Positive

Orphan designation: Elraglusib Treatment of pancreatic ...

Feb 20, 2025 0

Orphan designation: Elraglusib Treatment of pancreatic cancer, 16/01/2025 Positive

Orphan designation: Alvelestat Treatment of congenital alpha-1 antitrypsin deficiency, 16/01/2025 Positive

Orphan designation: Alvelestat Treatment of congenital ...

Feb 20, 2025 0

Orphan designation: Alvelestat Treatment of congenital alpha-1 antitrypsin defic...

Orphan designation: Camostat mesilate Treatment of chronic pancreatitis, 16/01/2025 Positive

Orphan designation: Camostat mesilate Treatment of chro...

Feb 20, 2025 0

Orphan designation: Camostat mesilate Treatment of chronic pancreatitis, 16/01/2...

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