The Pharma Data

NOVEMBER 2, 2020

Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) for vosoritide, an investigational, once daily injection analog of C-type Natriuretic Peptide (CNP) for children with achondroplasia, the most common form of disproportionate short stature in humans. (NASDAQ: BMRN) today announced that the U.S. In the U.S.,

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XTalks

DECEMBER 29, 2022

For example, on the pharma and biotech side, there has been growing demand for new drugs and biologics, from new classes of dual-action diabetes and weight loss drugs like Ozempic and Mounjaro to life-saving gene therapies. As of December 16, 2022, there are seven US Food and Drug Administration (FDA) approved gene therapies.

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pharmaphorum

OCTOBER 16, 2020

The repurposing of drugs is becoming more common, especially in the field of rare diseases. Now, as part of lifecycle management, pharmaceutical companies are looking more closely at drugs they have on their shelves. In the quest to repurpose a drug for a rare condition, there is a need to look at any and all available data.

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Delveinsight

AUGUST 17, 2021

Food and Drug Administration gave a red flag to FibroGen’s anemia pill roxadustat, for the use in patients suffering from kidney diseases owing to safety concerns that arose after results, which in turn are concluded for the presentation of further clinical studies to assess safety for future medicinal use.

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XTalks

APRIL 24, 2023

Rare diseases can often be progressive, chronic and fatal. Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Sadly, one-third of children with rare diseases die before their first birthday. How Can Study Protocols Be More Effective?

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The Pharma Data

NOVEMBER 8, 2020

Food and Drug Administration (FDA) in terms of PDUFA dates. The active ingredient is violaxazine hydrochloride, which has an extensive safety record in Europe, where it was previously marketed as an antidepressant. November is promising to be a busy month for the U.S. Supernus Pharmaceuticals’ SPN-812 for ADHD.

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The Pharma Data

DECEMBER 8, 2020

AbCellera’s AI-powered antibody discovery platform speeds the otherwise lengthy and grueling process by analyzing the database of natural immune systems to find antibodies that can be developed into drugs. Tackle the toughest problems in drug development.” The promise to partners is to “move quickly. Reduce cost. Remix Therapeutics .

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The Pharma Data

NOVEMBER 11, 2020

“ENPP1 deficiency is a systemic, progressive and continuous disease occurring over the course of a patient’s lifetime, starting as early as fetal development and spanning into adulthood. Received Rare Pediatric Disease Designation and Fast Track Designation from the U.S. Inozyme expects to share data from this study in 2021.

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XTalks

SEPTEMBER 17, 2020

Surrogate endpoints were used as the basis for approval of 45 percent of new drugs reviewed by the FDA between 2010 and 2012. For example, studies of heart attack prevention medications use the surrogate endpoint of low-density lipoprotein cholesterol (LDL-C) to assess the effectiveness of new drugs. Cushing’s Disease.

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XTalks

DECEMBER 13, 2023

Moreover, it has submitted global marketing authorization applications for mRNA-1345, a vaccine designed to prevent RSV-associated lower respiratory tract disease (RSV-LRTD) and acute respiratory disease (ARD), supported by favorable data from a predefined interim analysis. vaccine, mRNA-1273.815. million and $38.8

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Drug Discovery World

MARCH 6, 2024

To find new ways of diagnosing and treating complex diseases we first must understand the mechanisms underpinning their key pathological drivers, how these relate to different patient subgroups, and which drugs might be useful in ameliorating their effects – this is the basis of precision neuroscience. This is wholly unsurprising.

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The Pharma Data

AUGUST 3, 2021

Food and Drug Administration (FDA) has determined that OAV-101 intrathecal (IT) clinical trials for spinal muscular atrophy (SMA) patients may proceed, thereby lifting the partial clinical trial hold initiated in October 2019. Novartis today announced that the U.S.

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The Pharma Data

OCTOBER 14, 2020

is a clinical-stage biopharmaceutical company developing novel RNA-modulating drug candidates (designed to be eukaryotic ribosomal selective glycosides) that are formulated to treat rare and ultra-rare premature stop codon diseases. These premature stop codons have been identified in over 1,800 rare and ultra-rare diseases.

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Drug Discovery World

FEBRUARY 22, 2024

Lu Rahman looks at neuroscience drug discovery – advances and challenges – and how breakthroughs in this field are helping address areas of unmet need. Recent advances in biomarkers, research into the central nervous system and breakthroughs in human iPSC cell models, have bolstered neuroscience drug discovery research.

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The Pharma Data

OCTOBER 15, 2020

marketing and diagnostics. Sabbagh will be responsible for expanding Inozyme’s proprietary pipeline by identifying and developing new therapeutics for monogenic and non-genetic diseases of abnormal mineralization. Prior to joining Inozyme, Sabbagh served as the head of rare renal and musculoskeletal diseases research at Sanofi.

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The Pharma Data

OCTOBER 28, 2020

These SAEs were assessed as mild or moderate in severity and have generally improved over a period of a few weeks while disease domain improvements have been sustained for three months. The administration method will be modified to help reduce local drug contact time. Food and Drug Administration (FDA). About Ultragenyx.

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