The Pharma Data

AUGUST 9, 2021

Food and Drug Administration approved Nexviazyme (avalglucosidase alfa-ngpt) for intravenous infusion to treat patients 1 year of age and older with late-onset Pompe disease. “Pompe disease is a rare genetic disease that causes premature death and has a debilitating effect on people’s lives,” said Janet Maynard, M.D.,

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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

NOVEMBER 12, 2021

Treatment is First FDA-Approved Option Patients Can Take Regardless of Previous Therapies. Food and Drug Administration approved Besremi (ropeginterferon alfa-2b-njft) injection to treat adults with polycythemia vera, a blood disease that causes the overproduction of red blood cells. Today, the U.S.

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The Pharma Data

APRIL 19, 2022

Kite’s Global CAR T-Cell Therapy Manufacturing Network Increasing Capacity by 50% to Meet Patient Demand for New Cancer Therapies. — Scalable and Adaptable Facility Provides Flexibility for Current and Future Cell Therapy Innovation. The site will produce Kite’s FDA approved CAR T-cell therapy used to treat blood cancer.

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PLOS: DNA Science

OCTOBER 17, 2024

FDA approved a treatment for type 3 of the ultra-rare genetic disease September 20. The quest has been ongoing for three decades, said Laurie Turner, Family Services Manager of the National Niemann-Pick Disease Foundation. The new drug is taken with miglustat , which FDA approved for use in NPD3 in 2009.

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The Pharma Data

AUGUST 7, 2021

Approval is based on positive Phase 3 data demonstrating improvements in key disease burden measures and establishing its safety profile Nexviazyme specifically targets the M6P receptor, the key pathway for enzyme replacement therapy, to effectively clear glycogen build-up in muscle cells.

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Chemical Biology and Drug Design

OCTOBER 10, 2023

Abstract Human immunodeficiency virus (HIV) causes acquired immunodeficiency syndrome (AIDS), a lethal disease that is prevalent worldwide. Later, a new type of non-nucleoside reverse transcriptase inhibitors (NNRTIs) were approved as anti-HIV drugs. Molecular insights of HIV Reverse transcriptase and it's inhibitors.

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The Pharma Data

DECEMBER 17, 2020

Food and Drug Administration (FDA) has approved belimumab (Benlysta®), the first-ever treatment for adults with lupus nephritis (LN) who are currently receiving standard therapy. LN, or inflammation of the kidneys, is a common system of systemic lupus erythematosus (SLE), one of the most prevalent autoimmune diseases.

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The Pharma Data

APRIL 7, 2022

This unprecedented CMS decision effectively denies all Medicare beneficiaries access to ADUHELM ® (aducanumab-avwa), the first and only FDA-approved therapy in a new class of Alzheimer’s drugs. It may also limit coverage for any future approved treatment in the class. About Biogen.

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Fierce BioTech

SEPTEMBER 11, 2023

Aligning cross-functional teams across the organization to ensure medical, marketing, and market access are all in lock-step is even more crucial when launching a therapy into a rare disease market. Click here to login. Listing Image HerspiegelConsulting_LogoListingNew_10262023.png On Demand Start Date Thu, 10/26/2023 - 11:00

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Drug Target Review

FEBRUARY 21, 2024

Stem cell transplants have saved patients’ lives time and time again, which led us to launch our own Stem Cell Transplant and Cellular Therapy Program. Regarding CAR-T cell therapy, could you highlight the promising findings that have been identified in the early stages of drug discovery, as well as potential challenges?

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The Pharma Data

AUGUST 24, 2020

The FDA has approved a request from American Gene Technologies to begin a clinical study into its HIV gene therapy. The treatment is being researched by scientists collaborating from American Gene Technologies, the Laboratory of Immunoregulation and the National Institute of Allergy and Infectious Diseases.

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The Pharma Data

AUGUST 19, 2021

Food and Drug Administration (FDA) approval of the VENTANA MMR RxDx Panel, advancing the company’s commitment to personalised healthcare through tests that determine which patients are most likely to benefit from specific and targeted therapies. today announced U.S.

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Drug Target Review

AUGUST 29, 2024

What potential advantages does the CD5 modulation strategy offer over traditional CAR-T therapies? MR : Chimeric antigen receptor T-cell (CAR-T) therapy is very effective in treating patients with B-cell lymphoma, leukemia, and multiple myeloma, where we have six FDA-approved drugs.

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The Pharma Data

JANUARY 17, 2021

Food and Drug Administration (FDA) approved Janssen Pharmaceuticals ’ (a Johnson and Johnson company) Darzalex Faspro for adults with newly diagnosed light chain amyloidosis. It was approved in combination with bortezomib, cyclophosphamide and dexamethasone (D-VCd). Michael Vi/Shutterstock. It was developed with Genmab.

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KIF1A

MARCH 23, 2024

Each week, Dr. Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. We know that gene therapies are on the horizon, so it’s important to take note when groups make tangible progress.

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The Pharma Data

JUNE 22, 2023

FDA approval Pfizer (NYSE: PFE) announced that the U.S. 2) HRR gene mutations are found in approximately 25% of tumors from men with mCRPC and have been associated with aggressive disease and poor prognosis.(3,4,5,6) Therefore, new first-line treatment options are needed to reduce the risk of disease progression or death.

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The Pharma Data

AUGUST 27, 2020

Roche is committed to improve patient outcomes by providing multiple testing options that support decision-making during all lines of therapy. Cancer is a disease of the genome, driven by genetic mutations within a tumour’s DNA. Basel, 28 August 2020 – Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the U.S.

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The Pharma Data

SEPTEMBER 7, 2020

Genentech’s once-daily oral therapy Gavreto (pralsetinib) has secured FDA backing in the treatment of metastatic rearranged during transfection (RET) fusion-positive non-small cell lung cancer (NSCLC), it has emerged. In 27 treatment-naïve NSCLC patients, the ORR was found to be 70% and CRR was 11%.

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The Pharma Data

JANUARY 21, 2021

Food and Drug Administration (FDA) gave a greenlight for ViiV Healthcare ’s Cabenuva. Today’s FDA approval of Cabenuva represents a shift in the way HIV is treated, offering people living with HIV a completely new approach to care,” said Lynn Baxter, Head of North America, ViiV Healthcare. clinical practices.

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The Pharma Data

OCTOBER 14, 2021

Roche announced that gantenerumab, an anti-amyloid beta antibody developed for subcutaneous administration, has been granted Breakthrough Therapy Designation by the U.S. Food and Drug Administration (FDA) for the treatment of people living with Alzheimer’s disease (AD).

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Advarra

AUGUST 8, 2023

Gene therapy research is booming in the clinical setting. In this blog, we summarize the growth, risks, and regulatory requirements for gene therapy research. Defining the Boom in Gene Therapy Research The gene therapy field is experiencing explosive growth in today’s competitive research environment.

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The Pharma Data

AUGUST 31, 2021

today announced a label update for KEYTRUDA, Merck’s anti-PD-1 therapy, for its indication in first-line advanced urothelial carcinoma (bladder cancer) in the U.S. Food and Drug Administration (FDA) has converted this indication from an accelerated to a full (regular) approval. Source link: [link].

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The Pharma Data

OCTOBER 8, 2021

. “Today’s action marks the first FDA approval of a therapy to treat this very rare and devastating disease in children,” said Peter Marks, M.D., director of the FDA’s Center for Biologics Evaluation and Research. This application was granted a rare pediatric disease voucher by the FDA.

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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. NEW YORK, Nov. 25, 2020 (GLOBE NEWSWIRE) — Y-mAbs Therapeutics, Inc.

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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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The Pharma Data

DECEMBER 3, 2020

In the long-term APeX-S trial, those who completed 48 weeks of therapy had an average attack rate of 0.8 ORLADEYO is the first and only oral therapy that was designed to prevent attacks of HAE in adults and pediatric patients age 12 and older. attacks per month at baseline, to a mean of 1.0 attacks per month. attacks per month.

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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

AUGUST 10, 2020

Genentech’s Evrysdi (risdiplam) has secured FDA approval for the treatment of spinal muscular atrophy (SMA), making it the first oral therapy available for the rare hereditary genetic condition in US and the second overall to be approved for the disease. months at launch.

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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. Nasdaq:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic diseases of obesity, announced today that the U.S. BOSTON, Nov.

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The Pharma Data

MAY 5, 2021

Farxiga use expanded for reducing risks in patients with chronic kidney disease. Approval of the sodium-glucose cotransporter 2 (SGLT2) inhibitor was based on efficacy findings in the interventional, multicenter, randomized Dapa-CKD study. controlled trial had not received previous systemic therapy for metastatic disease.

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The Pharma Data

JUNE 28, 2021

Food and Drug Administration (FDA) granted Breakthrough Therapy designation for donanemab, Eli Lilly and Company’s (NYSE: LLY) investigational antibody therapy for Alzheimer’s disease (AD).

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The Pharma Data

AUGUST 21, 2020

The FDA has authorised Novartis’ Kesimpta (ofatumumab) for treating patients with relapsing forms of multiple sclerosis (MS), it has been revealed. . The injectable treatment also covers other forms of MS including clinically isolated syndrome and active secondary progressive disease. It can also be self-administered once a month.

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The Pharma Data

JANUARY 14, 2021

Food and Drug Administration (FDA) approved Pfizer ’s Xalkori (crizotinib) for pediatric patients one year of age and older and young adults with relapsed or refractory, systemic anaplastic large cell lymphoma (ALCL) that is anaplastic lymphoma kinase (ALK)-positive. Manuel Esteban/Shutterstock. Most Read Today. Source link.

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The Pharma Data

JANUARY 22, 2021

This approval will allow some patients the option of receiving once-monthly injections in lieu of a daily oral treatment regimen,” said Dr. John Farley, director of the Office of Infectious Diseases in the FDA’s Center for Drug Evaluation and Research.

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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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Vial

DECEMBER 11, 2023

Pathophysiological evidence links HCV infections to the risk of liver diseases such as hepatocellular carcinoma (HCC) and liver cirrhosis. The Food and Drug Administration (FDA) approved and recommended dozens of small-molecule drugs. Patients at advanced stages of liver disease can still be treated.

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