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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New Drug Approvals

NOVEMBER 13, 2023

PATENT WO2023214314 VODOBATINIB FOR REDUCING PROGRESSION OF PARKINSON’S DISEASE (wipo.int) Vodobatinib (N’-(2-chloro-6-methylbenzoyl)-4-methyl-3-[2-(3-quinolyl) ethynyl]-benzohydrazide), a c-Abl inhibitor is represented by Formula I (referred hereinafter interchangeably as vodobatinib or compound of Formula International Publication Nos.

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The Pharma Data

OCTOBER 26, 2020

(Nasdaq: PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that the U.S. In addition, the FDA has granted Fast Track designation for PR001 for the treatment of Parkinson’s disease with GBA1 mutations.

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The Pharma Data

MARCH 16, 2022

(Nasdaq: BIIB) announced that The Journal of Prevention of Alzheimer’s Disease (JPAD) today published a peer-reviewed manuscript detailing data from the pivotal Phase 3 EMERGE and ENGAGE trials for ADUHELM ® (aducanumab-avwa) 100 mg/mL injection for intravenous use in early Alzheimer’s disease.

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Alta Sciences

JUNE 27, 2023

Cancer Treatments, Clinical Trials, and the Motivation to Innovate nbartlett Tue, 06/27/2023 - 20:58 Getting to the Heart of Science with Michelle Newby At the heart of science are stories—stories of courage, challenges, successes, lessons, and people. Q: What was your cancer diagnosis and was treatment options were you given?

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The Pharma Data

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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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

MARCH 22, 2021

Basel, 22 March 2021 – Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced the decision to discontinue dosing in the Phase III GENERATION HD1 study of tominersen in manifest Huntington’s disease (HD). In December 2017, Roche licensed the investigational molecule from Ionis Pharmaceuticals. About Huntington’s disease.

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The Pharma Data

MARCH 16, 2022

These findings point to the potential of continued benefit of treatment in the longer term with continued reduction of amyloid beta plaques. These data demonstrate that long-term treatment with ADUHELM continues to reduce the underlying pathologies of Alzheimer’s disease beyond two years.” Biogen Inc.

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The Pharma Data

MARCH 2, 2021

Boehringer Ingelheim and Gubra today announced a new research and licensing agreement focused on the identification and validation of targets and innovative peptide compounds for the treatment of obesity. Senior Vice President and Global Head of Cardiometabolic Diseases Research, Boehringer Ingelheim. population will have obesity.

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Drug Target Review

AUGUST 16, 2023

Cancer remains a major global health concern, often diagnosed at advanced stages when treatment options and survival rates are limited. Furthermore, it successfully distinguished early-stage cancer samples from those with common diseases, laying the foundation for a robust data platform for further in-depth analysis. References 1.

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DNA Disease Therapies Treatment 98

Drug Target Review

OCTOBER 2, 2024

Cell and gene therapies (CGTs) have made significant advancements in treating oncological diseases, with therapies like CAR-T cell treatments transforming cancer care. This approach can be highly targeted, with the potential to correct mutations that lead to specific diseases or deliver therapeutic genes to improve heart function.

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The Pharma Data

JUNE 25, 2021

protofibril antibody for the treatment of Alzheimer’s disease (AD). 1 The proof-of-concept Study 201 explored the impact of treatment with lecanemab on reducing brain amyloid beta (A?) in individuals newly treated with lecanemab and was presented at the 2021 Alzheimer’s Disease and Parkinson’s Disease Conference.

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The Pharma Data

FEBRUARY 8, 2021

Janssen Announces Treatment with ERLEADA ® (apalutamide) Significantly Improved Overall Survival in Patients with Metastatic Castration-Sensitive Prostate Cancer. Based on these data, ADT alone should no longer be considered sufficient for patients with advanced, castration-sensitive disease.”. About ERLEADA ® (apalutamide).

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The Pharma Data

SEPTEMBER 29, 2021

In 2017, an alarming 1.2 to enhance outcomes, new treatments which will target kidney-specific disease mechanisms are highly desired,” said Hiddo L. to enhance outcomes, new treatments which will target kidney-specific disease mechanisms are highly desired,” said Hiddo L. 1 in 3 adults is at risk for the disease.

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Drug Target Review

JULY 10, 2023

The world needs better treatments for neurodegenerative diseases and other brain disorders. Despite decades of research and development, there are currently no drugs that can prevent or cure these diseases and only limited options for symptomatic relief. 3,4 Click or tap here to enter text.

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The Pharma Data

NOVEMBER 24, 2020

25, 2020 /CNW/ – On August 21, 2020 , Health Canada approved Lynparza® (olaparib), for the treatment of adult patients with deleterious or suspected deleterious germline and/or somatic BRCA or ATM- mutated metastatic castration-resistant prostate cancer (mCRPC) who have progressed following prior treatment with a new hormonal agent (NHA).

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The Pharma Data

FEBRUARY 22, 2021

Efanesoctocog alfa, a novel and investigational factor VIII therapy independent of von Willebrand Factor, is designed to provide near-normal factor activity levels for the majority of the week in a once-weekly prophylactic treatment regimen. Sanofi and Sobi collaborate on the development and commercialization of efanesoctocog alfa.

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The Pharma Data

JULY 21, 2021

Valneva SE (Nasdaq: VALN; Euronext Paris: VLA), a specialty vaccine company focused on the development and commercialization of prophylactic vaccines for infectious diseases with significant unmet medical need, and Pfizer Inc. Food and Drug Administration (FDA) in July 2017 3. About Lyme Disease. Juan Carlos Jaramillo M.D.,

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Drug Target Review

OCTOBER 17, 2023

To improve its treatment, early diagnosis and appropriate therapeutic strategies are very important. Metabolic changes and tumourigenicity Cancer is a multi-faceted disease which has previously been seen as a disorder in cell proliferation, the increase in cell numbers resulting from cell division. This research was published in Cell.

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Conversations in Drug Development Trends

AUGUST 10, 2023

As one of the more recent fields to emerge in the clinical space, psychedelics have gained significant traction as a potential treatment for various neuropsychiatric diseases. Psychedelic Research at Worldwide Since 2017, Worldwide Clinical Trials have supported a myriad of sponsors leading the way in psychedelic research programs.

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The Pharma Data

OCTOBER 29, 2020

If approved, aducanumab would become the first therapy to reduce the clinical decline of Alzheimer’s disease and to meaningfully change the course of Alzheimer’s disease. If approved, aducanumab would become the first therapy to reduce the clinical decline in patients with Alzheimer’s disease. CAMBRIDGE, Mass.

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The Pharma Data

NOVEMBER 6, 2020

The Advisory Committee also voted 0 yes, 7 no and 4 uncertain on the question, “Does Study 103 (PRIME) provide supportive evidence of the effectiveness of aducanumab for the treatment of Alzheimer’s disease?”, Aducanumab (BIIB037) is an investigational human monoclonal antibody studied for the treatment of Alzheimer’s disease.

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The Pharma Data

JUNE 7, 2022

based production in support of its COVID-19 oral treatment, PAXLOVID TM (nirmatrelvir [PF-07321332] tablets and ritonavir tablets). increasing Pfizer’s capability to produce and supply treatments and medicines for patients in the U.S. and has manufactured almost 17 million treatment courses total. and around the world.

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The Pharma Data

MARCH 9, 2022

The most common treatment-emergent adverse events (>5% of participants overall) were headache, arthralgia, fall, and back pain. Dietmar Berger, MD, PhD Global Head of Development and Chief Medical Officer , Sanofi “While advances have been made in the treatment of hemophilia, unmet medical needs still exist.

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The Pharma Data

JANUARY 3, 2021

Nasdaq: INZY), a clinical-stage biopharmaceutical company developing novel therapeutics for the treatment of rare diseases of abnormal mineralization impacting the vasculature, soft tissue and skeleton, today announced that the U.S. Inozyme Pharma was founded in 2017 by Joseph Schlessinger, Ph.D., . BOSTON, Jan.

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Clinical Trials Trials Treatment FDA 52

The Pharma Data

NOVEMBER 10, 2020

P otential to be a safer and effective alternative to the intravenous immunotherapies currently used for Crohn’s Disease. Crohn’s Disease Therapeutics Market Size $4.7 Oral and nasal administration routes are both physiologic approaches to stimulate the mucosal immune system to induce disease modifying benefits.”.

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Drug Target Review

JUNE 19, 2024

Our goal is to stand on the shoulders of giants and advance this exciting and new field of medicine, so that it can deliver valuable treatments to patients. There are many opportunities to address conditions of the eye, such as retinitis pigmentosa, dry age-related macular degeneration or any of the hundreds of inherited retinal diseases.

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The Pharma Data

MAY 18, 2021

Health Canada approves DUPIXENT® (dupilumab injection) as the first biologic for the treatment of moderate-to-severe atopic dermatitis in children aged 6 to 11 years. AD, the most common form of eczema, is an inflammatory disease classified by dry, itchy skin. MISSISSAUGA, ON , Feb. ” About DUPIXENT ®. .

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The Pharma Data

JANUARY 11, 2021

The agency previously granted the gene therapy Orphan Drug and Rare Pediatric Disease designations in 2017. Patients receiving the infusion did not achieve statistically significant muscle function following 48 weeks of treatment. Source link.

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The Pharma Data

AUGUST 9, 2020

Pivotal phase III stud y of etrolizumab in Crohn’s disease is ongoing. Roche is studying additional investigational medicines in inflammatory bowel diseases. In the HIBISCUS I induction study, in people without prior anti-tumour necrosis factor (anti-TNF) treatment, etrolizumab met the primary endpoint.

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Clinical Trials Disease Treatment Trials 52

The Pharma Data

SEPTEMBER 9, 2020

The company is testing higher doses of Ocrevus against both relapsing MS and primary progressive MS while also studying the treatment in minority populations. Roche is hoping to defend its franchise against MS treatments from its competitors. Conor Kavanagh. Source link.

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Trials Clinical Trials Treatment FDA 52

The Pharma Data

JUNE 18, 2021

In this study, patients who responded to 12 weeks of risankizumab intravenous (IV) induction treatment (in a prior study) were re-randomized to receive risankizumab 180 mg, risankizumab 360 mg or withdrawal from risankizumab treatment (risankizumab IV induction-only control group). NORTH CHICAGO, Ill. Deep Remission f. percent and 4.5

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The Pharma Data

NOVEMBER 2, 2020

adults by using blood pressure (BP) thresholds from the Seventh Report of the Joint National Committee on Prevention, Detection, Evaluation, and Treatment of High Blood Pressure (JNC7) and the 2017 American College of Cardiology-American Heart Association (ACC-AHA) BP guidelines. adults) using the 2017 ACC-AHA guideline criteria.

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Pharmaceuticals Treatment Disease Research 52

The Pharma Data

DECEMBER 3, 2020

The TAK-620-303 (SOLSTICE) trial (NCT02931539) is a multicenter, randomized, open-label, active-controlled trial comparing eight weeks of treatment with either maribavir or investigator assigned treatment (IAT) in transplant recipients with CMV infection refractory or resistant to existing antiviral treatments (i.e.,

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Treatment Pharmaceutical Companies Therapies Virus 52

Drug Target Review

OCTOBER 24, 2023

Live Bacterial Products (LBPs) will provide patients with a desirable, credible, safe and effective treatment option. The human microbiome, a complex ecosystem of microorganisms residing within our bodies, has emerged as a promising frontier in the quest for innovative medical treatments.

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Codon

JANUARY 21, 2024

Today, refined versions of these human challenge studies have become standard practice in testing vaccines for vector-borne diseases (e.g., yellow fever, malaria, and dengue), evaluating new drugs or treatments, and studying pathogenesis, the process by which a disease develops.

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