New Drug Approval

Blog Archives

The PI3K inhibitor Alpelisib (Piqray) for progressing breast cancer in women (and men)

Posted on May 30, 2019 by by

Joseph Gut - thasso Posted in Allelic Variant, New Drug Approval, Regulatory Approval, Targeted Therapy, Thasso Post, Theragenomic Medicine No Comments ↓

May 30, 2019 – The American Food and Drug Administration (FDA) recently approved Alpelisib (Piqray) to be used in combination with the FDA-approved endocrine therapy fulvestrant, to treat postmenopausal women, and men, with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative,

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Tags: Alpelisib (Piqray), American Food & Drug Administration (FDA), Breast Cancer, Companion Test, Fulvestrant (Faslodex), Male Breast Cancer, Phosphatidylinositol-4-5-bisphosphate 3-kinase catalytic subunit alpha (PIK3CA), PIK3CA Gene, PIK3CA Mutation, Stevens Johnson Syndrome [SJS], Thasso Post, Theragenomic Medicine, Therascreen PIK3CA RGQ PCR Kit, Toxic Epidermal Necrolysis (TEN)

Gene therapy: Zolgensma to treat spinal muscular atrophy

Posted on May 29, 2019 by by

Joseph Gut - thasso Posted in Children, Genetic Predisposition, New Drug Approval, Personalized Medicine, Targeted Therapy, Thasso Post, Theragenomic Medicine No Comments ↓

May 29, 2019 – Spinal muscular atrophy (SMA) is a group of neuromuscular disorders that result in the loss of motor neurons and progressive muscle wasting. The severity of symptoms and age of onset varies by the type. Some types are apparent at or before birth while others are not apparent until adulthood.

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Tags: American Food & Drug Administration (FDA), Gene Replacement Therapy, Gene Therapy, Infantile-Onset SMA, Onasemnogene Abeparvovec-xioi (Zolgensma), SMN1 Gene, Spinal Muscular Atrophy (SMA), Thasso Post, Theragenomic Medicine, Young Children, Zolgensma

New treatments for multiple sclerosis and inflammatory arthritis approved

Posted on March 31, 2019 by by

Joseph Gut - thasso Posted in New Drug Approval, Regulatory Approval, Thasso Post, Theragenomic Medicine 1 Comment ↓

March 31, 2019 – The American Food and Drug Administration (FDA) has just approved drugs for two rather problematic neurological conditions, namely multiple sclerosis (i.e., Cladribine (Mavenclad)) and non-radiographic axial spondyloarthritis (i.e., Certolizumab pegol (Cimzia)), offering patients with these conditions urgently need additional therapy options. FDA approves new oral treatment for multiple sclerosis

The FDA approved Cladribine (Mavenclad) tablets to treat relapsing forms of multiple sclerosis (MS ) in adults,

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Tags: American Food & Drug Administration (FDA), Boxed Warning, Certolizumab Pegol [Cimzia], Cladribine (Mavenclad), Multiple Sclerosis (MS), Non-Radiographic Axial Spondyloarthritis (nr-axSpA), Patient Medication Guide, Thasso Post, Theragenomic Medicine

Caplacizumab-yhdp (Cablivi): the first therapy for the treatment of aTTP

Posted on February 7, 2019 by by

Joseph Gut - thasso Posted in New Drug Approval, Rare Diseases, Thasso Post, Theragenomic Medicine 2 Comments ↓

February 07, 2019 – The American Food and Drug Administration (FDA) just approved Caplacizumab-yhdp (Cablivi), the first therapy specifically indicated, in combination with plasma exchange and immunosuppressive therapy, for the treatment of adult patients with acquired thrombotic thrombocytopenic purpura (aTTP),

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Tags: Acquired Thrombotic Thrombocytopenic Purpura (aTTP), ADAMTS13 Gene, American Food & Drug Administration (FDA), Caplacizumab-yhdp (Cablivi), Rare Disease, Thasso Post, Theragenomic Medicine, Upshaw–Schulman Syndrome, von Willebrand Factor (vWF)

Migalastat (Galafold): New treatment for Fabry disease

Posted on January 12, 2019 by by

Joseph Gut - thasso Posted in New Drug Approval, Rare Diseases, Thasso Post, Theragenomic Medicine No Comments ↓

January 12, 2018 – Fabry disease is an inherited disorder caused by mutations (alterations) in the alpha-galactosidase A (GLA) gene located on the X-chromosome. Fabry disease is rare and affects both males and females. It is estimated that classic Fabry disease (the most severe type) affects approximately one in 40,000 males.

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Tags: Alpha-Galactosidase A Gene (GLA), Fabry disease, Globotriaosylceramide (GL-3), Migalastat (Galafold), Rare Disease, Thasso Post, Theragenomic Medicine

Blastic plasmacytoid dendritic cell neoplasm (BPDCN): Tagraxofusp-erzs (Elzonris) FDA approved

Posted on December 30, 2018 by by

Joseph Gut - thasso Posted in New Drug Approval, Rare Diseases, Thasso Post, Theragenomic Medicine No Comments ↓

December 30, 2018 – Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is an aggressive and rare disease of the bone marrow and blood that can affect multiple organs, including the lymph nodes and the skin and often presents as or evolves into acute leukemia. The disease is more common in men than women and in patients 60 years and older.

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Tags: American Food & Drug Administration (FDA), Black Boxed Warning, Blastic Plasmacytoid Dendritic Cell Neoplasm, Capillary Leak Syndrome, Interleukin-3 Receptor (CD123), Life-threatening Adverse Effect, Rare Disease, Tagraxofusp-erzs (Elzonris), Thasso Post, Theragenomic Medicine

First ever treatment for Lambert-Eaton myasthenic syndrome (LEMS) FDA approved

Posted on December 4, 2018 by by

Joseph Gut - thasso Posted in New Drug Approval, Rare Diseases, Regulatory Announcement, Thasso Post, Theragenomic Medicine No Comments ↓

December 04, 2018 – The American Food and Drug Administration (FDA) has just approved its first ever treatment, Amifampridine (Firdapse), for Lambert-Eaton myasthenic syndrome (LEMS), a rare autoimmune disorder for adult patients. LEMS affects the connection between nerves and muscles and causes weakness and other symptoms in affected patients.

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Tags: American Food & Drug Administration (FDA), Amifampridine (Firdapse), Autoimmune Disorder, Lambert-Eaton Myasthenic Syndrome (LEMS), Rare Disease, Thasso Post, Theragenomic Medicine

Hope for patients with cutaneous squamous cell carcinoma (CSCC): Cemiplimab-Rwlc (Libtayo)

Posted on October 30, 2018 by by

Joseph Gut - thasso Posted in New Drug Approval, Personalized Medicine, Targeted Therapy, Thasso Post, Theragenomic Medicine No Comments ↓

October 31, 2018 – The American Food and Drug Administration (FDA) recently approved Cemiplimab-Rwlc (Libtayo) injection for intravenous use for the treatment of patients with metastatic cutaneous squamous cell carcinoma (CSCC) or locally advanced CSCC who are not candidates for curative surgery or curative radiation.

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Tags: Autoimmunity, Cemiplimab-Rwlc (Libtayo), Food and Drug Administration (FDA), Immune Checkpoint Inhibitors (ICI's), Metastatic Cutaneous Squamous Cell Carcinoma (CSCC), Targeted Therapy, Thasso Post, Theragenomic Medicine

Patisiran (Onpattro): First targeted RNA-based therapy approved to treat hATTR

Posted on August 13, 2018 by by

Joseph Gut - thasso Posted in Genetic Background, New Drug Approval, Rare Diseases, Targeted Therapy, Thasso Post, Theragenomic Medicine No Comments ↓

August 14, 2018 – The American Food and Drug Administration (FDA) just approved Patisiran (Onpattro) infusion for the treatment of peripheral nerve disease (polyneuropathy) caused by hereditary transthyretin-mediated amyloidosis (hATTR) in adult patients.

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Tags: Hereditary Transthyretin-Mediated Amyloidosis (hATTR), Patisiran (Onpattro), siRNA Treatment, Small Interfering Ribonucleic Acid (siRNA), Targeted Therapy, Thasso Post, Theragenomic Medicine

Ivosidenib (Tibsovo) for AML-patients with IDH1-mutations

Posted on July 22, 2018 by by

Joseph Gut - thasso Posted in Allelic Variant, Companion Test, New Drug Approval, Personalized Medicine, Regulatory Approval, Targeted Therapy, Thasso Post, Theragenomic Medicine No Comments ↓

July 22, 2018 – The American Food and Drug Administration (FDA) just approved Ivosidenib (Tibsovo) for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) who have a specific genetic mutation in the IDH1 gene.

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