The Pharma Data

JUNE 9, 2025

Xywav: A Low-Sodium Alternative with FDA Approval Xywav is a uniquely formulated, low-sodium oxybate therapy, and remains the only product of its kind approved by the U.S. It is also approved for adult patients with idiopathic hypersomnia (IH).

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SugarCone Biotech

JUNE 19, 2025

Finally, while there are clear front runners among the pharmaceutical companies developing targeted therapeutics for these antigens there are also emerging biotechs aggressively pursuing these targets. Over 70% of new patients are diagnosed with stage IV disease, also called extensive stage disease (ES-SCLC).

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The Pharma Data

AUGUST 27, 2020

Cancer is a disease of the genome, driven by genetic mutations within a tumour’s DNA. The convenience of testing a blood sample may also enable more rapid treatment decisions, so that patients can feel reassured they are not losing time to fight their disease.”.

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New Drug Approvals

MAY 15, 2025

5] Leniolisib was approved for medical use in the United States in March 2023. [5] 5] [7] [8] It is the first approved medication for the treatment of activated PI3K delta syndrome. [5] 5] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication. [9] “Leniolisib: First Approval” Drugs.

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The Pharma Data

NOVEMBER 27, 2020

FDA Approves Imcivree (setmelanotide) for Chronic Weight Management in Patients with Obesity Due to POMC, PCSK1 or LEPR Deficiency. 27, 2020 (GLOBE NEWSWIRE) — Rhythm Pharmaceuticals, Inc. With this approval, Imcivree becomes the first-ever FDA approved therapy for these rare genetic diseases of obesity.

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The Pharma Data

DECEMBER 16, 2020

17, 2020 /PRNewswire/ — CNS Pharmaceuticals, Inc. We will now rapidly move to initiate our Phase 2 trial of Berubicin for adults with GBM and expect to begin enrolling patients in the first quarter of next year,” commented John Climaco , CEO of CNS Pharmaceuticals. About CNS Pharmaceuticals, Inc.

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The Pharma Data

NOVEMBER 27, 2020

FDA Approves Danyelza (naxitamab-gqgk) for the Treatment of Neuroblastoma. This indication is approved under accelerated approval regulation based on overall response rate and duration of response. This indication is approved under accelerated approval based on overall response rate and duration of response.

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Metabolite Tales Blog

JANUARY 26, 2023

Metabolism of 2022 FDA approved small molecule drugs – Part 1 Does CYP3A4 still rule? By Julia Shanu-Wilson It won’t come as much surprise to learn that of the 17 small molecules* approved by the FDA in 2022, CYP3A4 was the major player in drug metabolism. References Iversen et al., Curr Drug Metab. 20(4): 254-265.

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The Pharma Data

OCTOBER 26, 2020

Kala Pharmaceuticals announced today that the U.S. Food and Drug Administration (FDA) has approved EYSUVIS for the short-term treatment of dry eye disease. . adults have been diagnosed with dry eye disease, a chronic, episodic, multifactorial disease. Roughly 16.4 million U.S.

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The Pharma Data

JUNE 11, 2025

Ionis Begins Pivotal Phase 3 REVEAL Study of ION582 in Angelman Syndrome, Dosing First Patient in Global Trial Ionis Pharmaceuticals , Inc. This trial will build on previous positive data from the earlier Phase 1/2 HALOS study and is designed to rigorously assess the potential of ION582 as a disease-modifying therapy. “We Kordasiewicz.

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The Pharma Data

DECEMBER 1, 2020

VICTORIA, British Columbia–( BUSINESS WIRE )– Aurinia Pharmaceuticals Inc. Aurinia Pharmaceuticals is a late-stage clinical biopharmaceutical company focused on developing and commercializing therapies to treat targeted patient populations that are impacted by serious diseases with a high unmet medical need.

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The Pharma Data

DECEMBER 15, 2020

FDA Approves Klisyri (tirbanibulin) for the Treatment of Actinic Keratosis on the Face or Scalp. Food and Drug Administration (FDA) has approved Klisyri (tirbanibulin) for the topical treatment of actinic keratosis (AK) on the face or scalp. The FDA approval of Klisyri is a significant milestone for Athenex.

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The Pharma Data

MAY 27, 2022

Novartis today announced the US Food and Drug Administration (FDA) has granted accelerated approval for Kymriah ® (tisagenlecleucel) for the treatment of adult patients with relapsed or refractory (r/r) follicular lymphoma (FL) after two or more lines of systemic therapy.

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The Pharma Data

JANUARY 7, 2021

VICTORIA, British Columbia–( BUSINESS WIRE )– Aurinia Pharmaceuticals Inc. Aurinia Pharmaceuticals is a late-stage clinical biopharmaceutical company focused on developing and commercializing therapies to treat targeted patient populations that are impacted by serious diseases with a high unmet medical need.

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Advarra

AUGUST 16, 2022

A rare, or orphan, disease by definition affects a small percentage of the population — fewer than 200,000 people in the U.S. But the numbers add up, and taken together, rare diseases impact an estimated 30 million Americans. Food and Drug Administration (FDA) approval. The FDA Since 1983. FDA Expedited Programs.

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The Pharma Data

APRIL 19, 2022

Food and Drug Administration (FDA) has approved commercial production at the company’s new CAR T-cell therapy manufacturing facility in Frederick, Maryland. The site will produce Kite’s FDA approved CAR T-cell therapy used to treat blood cancer. Gilead Sciences, Inc. Source link: [link].

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New Drug Approvals

JULY 8, 2025

Salt Formation: The pritelivir free base is then reacted with methanesulfonic acid to form the mesylate salt, which is the active pharmaceutical ingredient (API). 7] If the virus also acquires resistance to foscarnet, then there is currently no FDA approved treatment.

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The Pharma Data

AUGUST 31, 2020

Detects, confirms and differentiates HIV-1 and HIV-2 infections providing clinicians with critical diagnostic data for personalised management of patients with HIV – from appropriate counseling on disease differences to targeted therapy. Centers for Disease Control and Prevention (CDC) estimated in 2018 that 1.2

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DrugBank

DECEMBER 18, 2024

A new shift is occurring in the pharmaceutical industry, leading to a rapidly expanding field known as digital therapeutics (DTx). These interventions target a broad spectrum of medical conditions, encompassing chronic diseases and even substance abuse.

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KIF1A

JULY 1, 2023

Each week, Dr. Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. But promising drugs that didn’t make the cut for treating one disorder may still be effective for other rare diseases. What’s a pre-print?

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The Pharma Data

JANUARY 5, 2021

RAD011 is a pivotal-trial ready synthetic cannabidiol oral solution with potential utilization in multiple endocrine and metabolic orphan diseases. Prader-Willi syndrome (“PWS”) will be the initial indication, which has been granted Orphan Drug and Fast Track Designation by the FDA. Disease Highlights.

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FDA Law Blog: Biosimilars

JUNE 27, 2023

Amongst his accomplishments, Law360 considered the role James has played in leveraging little-used pathways to FDA approval for often first-ever drugs to treat rare diseases (e.g., Duchenne, Friederichs’s Ataxia, Sickle Cell Disease, Chagas, ALS).

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New Drug Approvals

APRIL 25, 2025

3] Inavolisib was approved for medical use in the United States in October 2024. [3] 19] Society and culture Legal status In October 2024, the US Food and Drug Administration (FDA) approved inavolisib for the treatment of PIK3CA -mutant breast cancer based on the results from the INAVO120 trial. [3] 3 November 2006. PMC 8706051.

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The Pharma Data

OCTOBER 14, 2021

Food and Drug Administration (FDA) for the treatment of people living with Alzheimer’s disease (AD). “This Breakthrough Therapy Designation reinforces our confidence in gantenerumab, which would be the first subcutaneous medicine for the treatment of Alzheimer’s disease with the potential for at-home administration.

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The Pharma Data

DECEMBER 3, 2020

BioCryst Pharmaceuticals, Inc. Food and Drug Administration (FDA) has approved ORLADEYO (berotralstat) for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adults and young patients 12 years and older. announced on Thursday that the U.S.

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The Pharma Data

JULY 8, 2021

ADUHELM should be initiated in patients with mild cognitive impairment due to Alzheimer’s disease or mild Alzheimer’s dementia. Food and Drug Administration (FDA) has approved an updated label for ADUHELM (aducanumab-avwa) injection 100 mg/mL solution. ADUHELM is indicated for the treatment of Alzheimer’s disease.

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The Pharma Data

MARCH 5, 2021

Food and Drug Administration (FDA) approved Actemra ® /RoActemra ® (tocilizumab) subcutaneous injection for slowing the rate of decline in pulmonary function in adult patients with systemic sclerosis-associated interstitial lung disease (SSc-ILD), a debilitating condition with limited treatment options.

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The Pharma Data

JANUARY 6, 2021

SK Chemicals has shared their expertise in these diseases and validated the predicted targets and compounds through in vitro and in vivo studies. We will continue to expand our collaboration to address unmet needs in treating intractable diseases.”. About Standigm. Standigm is an AI-driven drug discovery company.

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Drug Target Review

SEPTEMBER 19, 2023

The chips can be modelled with primary cells, IPS derived stem cells or patient derived cells and potentiates to unlock molecular mechanisms that drive the disease pathophysiology, holding a greater promise. These guidelines drive the legitimacy of the models towards regulatory approval.

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The Pharma Data

DECEMBER 15, 2020

A healthy immune system defends the body against disease and other conditions. Autoimmune disease impacts different parts of the body, weakening functionality. Researchers are aware of more than 80 diseases that occur when the immune system attacks the body’s own organs, tissues and cells. It may be life-threatening.

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The Pharma Data

NOVEMBER 26, 2020

(NYSE American: PLX) (TASE: PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx ® plant cell-based protein expression system, and Chiesi Global Rare Diseases, a business unit of Chiesi Farmaceutici S.p.A.,

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The Pharma Data

DECEMBER 16, 2020

16, 2020 /PRNewswire/ — Regeneron Pharmaceuticals, Inc. About Regeneron Regeneron (NASDAQ: REGN) is a leading biotechnology company that invents life-transforming medicines for people with serious diseases. SOURCE Regeneron Pharmaceuticals, Inc. TARRYTOWN, N.Y. , Morgan Healthcare Conference on Monday, January 11, 2021.

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The Premier Consulting Blog

JANUARY 14, 2025

Provides the Oncology Accelerated Approvals Database. Project Frontrunner An initiative for the development of new therapies to the early clinical setting Develop a framework for identifying drug candidates that are appropriate to initially develop for early metastatic disease (e.g.,

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The Pharma Data

JANUARY 19, 2021

19, 2021 /PRNewswire/ — Regeneron Pharmaceuticals, Inc. About Regeneron Regeneron (NASDAQ: REGN) is a leading biotechnology company that invents life-transforming medicines for people with serious diseases. SOURCE Regeneron Pharmaceuticals, Inc. TARRYTOWN, N.Y. , financial markets open. View original content: [link].

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The Pharma Data

NOVEMBER 30, 2020

30, 2020 /PRNewswire/ — Regeneron Pharmaceuticals, Inc. About Regeneron Regeneron (NASDAQ: REGN) is a leading biotechnology company that invents life-transforming medicines for people with serious diseases. SOURCE Regeneron Pharmaceuticals, Inc. TARRYTOWN, N.Y. , View original content: [link]. Source link.

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The Pharma Data

OCTOBER 27, 2020

27, 2020 /PRNewswire/ — Regeneron Pharmaceuticals, Inc. About Regeneron Regeneron (NASDAQ: REGN) is a leading biotechnology company that invents life-transforming medicines for people with serious diseases. SOURCE Regeneron Pharmaceuticals, Inc. TARRYTOWN, N.Y. , EST on Monday, November 9, 2020. Source link.

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New Drug Approvals

APRIL 18, 2025

2] Crinecerfont was approved for medical use in the United States in December 2024. [2] 2] [3] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication. [4] 2] Crinecerfont was approved for medical use in the United States in December 2024. [2] Food and Drug Administration (FDA) (Press release).

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