Drug Target Review

JUNE 21, 2023

Most rare diseases are caused by a single gene defect, but severity can vary considerably among patients. Modifier genes can help explain that variability and can alter or even prevent disease onset and progression, making them appealing therapeutic targets. However, the identification of these genes is challenging.

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FDA Law Blog: Drug Discovery

DECEMBER 13, 2023

Sasinowski — On December 12, 2023, FDA announced the creation of a new advisory committee specifically for treatments for genetic metabolic diseases, the Genetic Metabolic Diseases Advisory Committee, or “GeMDAC.” There are hundreds of known genetic metabolic diseases, most of which are rare and carry significant morbidity.

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DrugBank

JULY 20, 2023

of the global population) suffer from rare conditions without effective treatments or cures. Rare diseases, therefore, present compelling opportunities for Drug Development. Since each disease is present in small groups of patients, this research is also fraught with complex challenges. At any given time, millions of people ( 3.5–5.9%

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The Pharma Data

APRIL 7, 2022

It may also limit coverage for any future approved treatment in the class. These coverage restrictions, including the distinction between accelerated approval and traditional approval, have never been applied to FDA-approved medicines for other disease areas. ADUHELM is indicated for the treatment of Alzheimer’s disease.

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The Pharma Data

NOVEMBER 9, 2020

An experimental Alzheimer’s disease treatment should not be approved by the U.S. The drug, developed by the pharmaceutical company Biogen and its Japanese partner Eisai, is administered through intravenous infusion for early Alzheimer’s disease. © 2020 HealthDay. Posted: November 2020. Source link.

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The Pharma Data

NOVEMBER 26, 2020

27, 2020 /PRNewswire/ — Protalix Biotherapeutics, Inc. Priority Review is granted to therapies that the FDA determines have the potential to provide significant improvements in the treatment, diagnosis or prevention of serious conditions. About Chiesi Global Rare Diseases.

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The Pharma Data

AUGUST 15, 2021

Food and Drug Administration (FDA) has granted Fast Track designation to VTX-801, Vivet’s clinical-stage gene therapy for the treatment of Wilson Disease – a rare, genetic disorder that reduces the ability of the liver and other tissues to regulate copper levels, causing severe hepatic damage, neurological symptoms, and potentially death.

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The Pharma Data

DECEMBER 29, 2020

30, 2020 /PRNewswire/ — Protalix BioTherapeutics, Inc. an international research-focused healthcare Group (Chiesi Group), today announced final study results from the BRIDGE Phase III Open-Label, Switch-Over Clinical Trial Evaluating Pegunigalsidase Alfa for the Treatment of Fabry Disease.

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The Pharma Data

JUNE 29, 2021

If approved, Forxiga has the potential to change the treatment paradigm for millions of people in the EU suffering from chronic kidney disease. CKD is a serious, progressive condition defined by decreased kidney function and is often associated with an increased risk of heart disease or stroke.

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The Pharma Data

DECEMBER 30, 2020

The year 2020 will certainly go down in history as the year of COVID-19. Gilead Sciences Makes Multiple Bets : When Gilead Sciences wasn’t working on remdesivir in COVID-19, the company seemed to be constantly flexing its M&A muscle over the course of 2020. It began in May with the $4.9 But the U.K.-based

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New Drug Approvals

SEPTEMBER 29, 2024

gingivalis has been called a risk factor for Alzheimer’s disease ( Kanagasingam et al., gingivalis is associated with cardiovascular disease. Both were reversed by treatment with COR388 ( 2020 AAIC abstract ). gingivalis DNA and gingipains in CSF, blood, and saliva, before and after treatment. and Europe.

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The Pharma Data

MARCH 28, 2022

Kerendia ™ (10 mg or 20 mg), a non-steroidal, selective mineralocorticoid receptor (MR) antagonist, is approved for the treatment of chronic kidney disease and type 2 diabetes, excluding patients with end-stage renal disease or on dialysis. Finerenone is different to existing CKD in T2D treatments.

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The Pharma Data

JULY 12, 2021

Finerenone 10 mg or 20 mg is indicated to reduce the risk of sustained estimated glomerular filtration rate (eGFR) decline, end-stage kidney disease, cardiovascular death, non-fatal myocardial infarction, and hospitalization for heart failure in adult patients with chronic kidney disease (CKD) associated with type 2 diabetes (T2D).

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The Pharma Data

NOVEMBER 8, 2020

Verona Pharma is a clinical-stage biopharmaceutical company focused on developing and commercializing innovative therapies for the treatment of respiratory diseases with significant unmet medical needs. LONDON and RALEIGH, N.C., For more information, please visit www.veronapharma.com. Forward-Looking Statements.

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The Pharma Data

OCTOBER 20, 2020

21, 2020 /PRNewswire/ — Ionis Pharmaceuticals, Inc. Eastern Time to discuss its third quarter 2020 financial results and report on pipeline and business progress. Webcast scheduled for Wednesday, November 4 at 11:30 a.m. Eastern Time. . CARLSBAD, Calif. , Interested parties may access the webcast at www.ionispharma.com.

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The Pharma Data

OCTOBER 26, 2020

27, 2020 (GLOBE NEWSWIRE) — Prevail Therapeutics Inc. Nasdaq: PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that the U.S. NEW YORK, Oct. Founder and Chief Executive Officer of Prevail. “FDA

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The Premier Consulting Blog

JUNE 18, 2023

One exciting application of these technologies is the use of in silico trials in the development of novel therapies for rare diseases. However, with rare diseases there may be no available treatments that could serve as SoC or active control in a clinical trial and assigning patients to placebo may be unethical.

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The Pharma Data

NOVEMBER 18, 2020

Antimicrobial resistance occurs when microorganisms resist the effects of medications, making infections harder to treat and increasing the risk of disease spread, severe illness and death. Key definitions. Combatting antimicrobial resistance is a global. The strategy provides an overview of WHO-recommended. taking PrEP.

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The Pharma Data

JANUARY 12, 2021

(NASDAQ: NARI) (“Inari”) a commercial-stage medical device company focused on developing products to treat and transform the lives of patients suffering from venous diseases, today reported preliminary unaudited fourth quarter 2020 revenue. million to $48.9 million to $48.9 About Inari Medical, Inc. Inari Medical, Inc.

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The Pharma Data

JUNE 7, 2022

AL01811 is a preclinical selective GBA2 inhibitor with first-in-class potential as an oral disease modifying treatment for Parkinson’s Disease Alectos to receive a $15 million upfront payment and is eligible to receive potential future development and commercial milestone payments. Biogen Inc.

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The Pharma Data

DECEMBER 31, 2020

Monthly News Roundup – December 2020. In studies, the vaccines were found to be 94% to 95% effective in preventing COVID-19 disease. The partial piece of the spike protein cannot cause COVID-19 disease. Moderna and Pfizer’s COVID-19 Vaccines Roll Out in Early Phase. In December, the U.S. How do they work?

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The Pharma Data

DECEMBER 30, 2020

The Feverish Pricing of MS Treatments. Towards the end of March, the FDA approved two new treatments for multiple sclerosis (MS): Mayzent (siponimod) and Mavenclad (cladribine). Drug treatments for MS have been climbing an expensive staircase for a number of years. Posted: December 2020. Consumer News. Source link.

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The Pharma Data

SEPTEMBER 28, 2020

Although Chagas disease was discovered more than a century ago, it remains a major public health challenge. Chagas disease affects approximately six million people, mainly in Latin America. Like other neglected tropical diseases, Chagas disease often affects poor and marginalized communities. Chagas Disease.

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The Pharma Data

NOVEMBER 30, 2020

30, 2020 /PRNewswire/ — Regeneron Pharmaceuticals, Inc. NASDAQ: REGN) will host a webcast on Monday, December 7, 2020 at 4:30pm EST. Management will discuss data presented at the American Society of Hematology (ASH) 2020 Annual Meeting as well as provide updates on the Company’s broader oncology and hematology portfolio.

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The Pharma Data

OCTOBER 27, 2020

28, 2020 /PRNewswire/ — ReVision Therapeutics, Inc., 28, 2020 /PRNewswire/ — ReVision Therapeutics, Inc., Stargardt disease is a devastating genetic disorder that affects central vision with no approved treatment options. . RIDGEWOOD, N.J. , About reVision Therapeutics, Inc.

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The Pharma Data

OCTOBER 27, 2020

28, 2020 (GLOBE NEWSWIRE) — Passage Bio, Inc. Food and Drug Administration (FDA) has granted Orphan Drug and Rare Pediatric Disease (RPD) designations to PBKR03 for the treatment of Krabbe disease (Globoid Cell Leukodystrophy). About Krabbe Disease. PHILADELPHIA, Oct.

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New Drug Approvals

SEPTEMBER 23, 2024

Target: DNA/RNA Synthesis Pathway: Cell Cycle/DNA Damage Huntington’s disease (HD) is a progressive, autosomal dominant neurodegenerative disorder of the brain, having symptoms characterized by involuntary movements, cognitive impairment, and mental deterioration. 2020-01-02. SCHEME PATENT PTC Therapeutics Inc., Audiol Res. J Pers Med.

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Drug Target Review

JULY 1, 2024

As a non-opioid, SRP-001 also eliminates abuse potential, positioning it as a safer and effective drug candidate for the treatment of acute and neuropathic pain and migraine headache. How does SRP-001’s safety profile, especially concerning hepatotoxicity and nephrotoxicity, compare to other common pain medications?

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The Pharma Data

OCTOBER 15, 2020

Vertex Pharmaceuticals has decided to give up on its experimental VX-814, a small molecule drug for the rare genetic disease Alpha-1 antitrypsin deficiency (AATD), canning the drug’s development after seeing lackluster results from an early phase 2 trial. James Miessler.

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The Pharma Data

NOVEMBER 25, 2020

AHA: Ablation Better Than Meds for Initial A-Fib Treatment. 25, 2020 — Cryoballoon ablation is superior to drug therapy as an initial treatment for the prevention of atrial arrhythmia recurrence in patients with paroxysmal atrial fibrillation , according to a study published online Nov. © 2020 HealthDay. Wazni, M.D.,

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The Pharma Data

DECEMBER 17, 2020

18, 2020 /PRNewswire/ — Camurus AB (NASDAQ STO: CAMX) announced today that the Swiss agency for therapeutic products, Swissmedic, has approved weekly and monthly Buvidal ® prolonged release buprenorphine for the treatment of opioid dependence in adults and adolescents from 16 years of age. LUND, Sweden , Dec. About Buvidal.

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The Pharma Data

AUGUST 19, 2020

August 19, 2020 – Johnson & Johnson (NYSE:JNJ) today announced it has entered into a definitive agreement to acquire Momenta Pharmaceuticals, Inc. Momenta), a company that discovers and develops novel therapies for immune-mediated diseases, in an all cash transaction for approximately $6.5 NEW BRUNSWICK, N.J., An estimated 2.5

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The Pharma Data

DECEMBER 28, 2020

Triple Treatment Combo Beneficial in BRAF-Mutant CRC. 28, 2020 — Irinotecan and cetuximab combined with vemurafenib improve progression-free survival in BRAF V600E -mutated colorectal cancer (CRC), according to a study published online Dec. © 2020 HealthDay. Posted: December 2020. Professional. MONDAY, Dec.

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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. Since then, the FDA has significantly changed its approach to rare and orphan diseases.

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The Pharma Data

DECEMBER 4, 2020

Alzheimer Disease Tied to Adverse Financial Events. 4, 2020 — Alzheimer disease and related dementias (ADRDs) are associated with adverse financial events years prior to clinical diagnosis, according to a study published online Nov. © 2020 HealthDay. Posted: December 2020. Professional. FRIDAY, Dec.

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The Pharma Data

OCTOBER 18, 2020

19, 2020 06:00 UTC. ZURICH, Switzerland–( BUSINESS WIRE )– Versantis AG , a clinical-stage company developing novel therapies for orphan liver diseases, today announced that the U.S. Current treatment options are associated with poor outcomes. © 2020, Versantis AG, all rights reserved. About Versantis.

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DECEMBER 18, 2020

18 December 2020 — The US Food and Drug Administration (FDA) has requested further clarifying analyses of clinical data, to complete its review of the New Drug Application (NDA) for roxadustat, an oral hypoxia-inducible factor prolyl hydroxylase (HIF-PH) inhibitor for patients with anemia of chronic kidney disease (CKD).

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