The Pharma Data

JUNE 2, 2023

Food and Drug Administration (FDA) for the treatment of adult patients with deleterious or suspected deleterious BRCA -mutated ( BRCA m) metastatic castration-resistant prostate cancer (mCRPC). Patients should be selected for therapy based on an FDA-approved companion diagnostic for LYNPARZA. In the U.S., For the U.S.

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PLOS: DNA Science

JUNE 1, 2023

The newest FDA-approved gene therapy treats the severe, skin-peeling condition dystrophic epidermolysis bullosa (DEB). The gene treatment has been a long time coming, but it differs from the handful of other approved gene therapies: it isn’t a one-and-done. Earlier results were published in Nature Medicine.

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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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The Pharma Data

AUGUST 24, 2020

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. The Phase 1 trial is expected to be conducted at sites in Baltimore, Maryland and Washington, DC.

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The Pharma Data

JANUARY 21, 2021

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. The approval was built on the Phase III ATLAS and FLAIR trials. Janssen is a Johnson & Johnson company.

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Drug Target Review

JANUARY 8, 2024

It is no mystery that as we age our health starts to deteriorate, and we become increasingly susceptible to diseases. Through years of scientific exploration and research, we now know there are several biological changes that make our bodies more susceptible to disease or injury, and we can target those with therapeutic interventions.

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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

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. The FDA granted approval under the accelerated approval regulation. NEW YORK, Nov.

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The Pharma Data

SEPTEMBER 7, 2020

While the approval has been awarded, the FDA warned that continued approval may be dependent on Genentech reinforcing these positive data with a future confirmatory clinical trial. These benefits were seen in patients regardless of RET fusion partner or central nervous system involvement. Matt Fellows. Source link.

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FDA Law Blog: Drug Discovery

MARCH 26, 2024

s Frank Sasinowski , James Valentine and Charles Raver are honored to have aided Italfarmaco in the development and approval of this new drug, and to be part of the effort to expand treatment options for the young men and boys living with Duchenne, and the families of those affected by Duchenne. Hyman, Phelps & McNamara, P.C.’s

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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

OCTOBER 22, 2020

FDA Approves Veklury (remdesivir) for the Treatment of COVID-19. Food and Drug Administration (FDA) has approved the antiviral drug Veklury (remdesivir) for the treatment of patients with COVID-19 requiring hospitalization. The speed and rigor with which Veklury has been developed and approved in the U.S.

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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

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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The Pharma Data

AUGUST 31, 2021

10), as determined by an FDA-approved test, or in patients who were not eligible for any platinum-containing chemotherapy regardless of PD-L1 status. This indication was approved under accelerated approval based on tumor response rate and duration of response.

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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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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

JANUARY 11, 2021

based subsidiary of Terumo and a global neurovascular company, announced today the FDA Approval of the PMA Supplement for the WEB 17 System, a new addition to the WEB Aneurysm Embolization System for the treatment of intracranial wide neck bifurcation aneurysms. . ALISO VIEJO, Calif. ,

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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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Vial

JUNE 12, 2024

PROs in clinical trials are important as they capture the patient’s perspective and ensure that the impact of an intervention is comprehensively evaluated. Food and Drug Administration (FDA) increasingly look to patients to understand how they describe their health status. What are PROs in clinical trials?

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Conversations in Drug Development Trends

FEBRUARY 26, 2024

With the necessary prioritization of various factors when designing your clinical trial, the patient experience can be overlooked or improperly addressed in study design. It plays a significant role in patient recruitment and retention, both of which are vital to maintaining timelines, decreasing program costs, and gaining market approval.

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The Pharma Data

JUNE 26, 2023

FDA approves Pfizer’s LITFULO™ (ritlecitinib) for adults and adolescents with severe alopecia areata Pfizer Inc. Food and Drug Administration (FDA) has approved LITFULO™ (ritlecitinib), a once-daily oral treatment, for individuals 12 years of age and older with severe alopecia areata. Source link: [link]

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The Pharma Data

OCTOBER 26, 2020

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. EYSUVIS is the first FDA-approved corticosteroid specifically for dry eye disease treatment.

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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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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

SEPTEMBER 28, 2020

This approval comes after GSK submitted results from its Phase 3 trial which enrolled 108 patients. Today’s approval gives these patients access to biologic treatment for the first time and demonstrates our commitment to maximising Nucala’s impact on eosinophil-driven diseases. “.

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The Pharma Data

NOVEMBER 16, 2020

November 16, 2020 — An independent data and safety monitoring board (DSMB) overseeing the Phase 3 trial of the investigational COVID-19 vaccine known as mRNA-1273 reviewed trial data and shared its interim analysis with the trial oversight group on Nov. 37% of trial volunteers are from racial and ethnic minorities.

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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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The Pharma Data

DECEMBER 29, 2020

Phase III BRIDGE open-label, switch-over clinical trial met key objectives for safety and efficacy. galactosidase-A product candidate under development for the treatment of Fabry disease. CARMIEL, Israel and BOSTON , Dec. 30, 2020 /PRNewswire/ — Protalix BioTherapeutics, Inc. Male patients improved from -6.36

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Vial

DECEMBER 5, 2023

Among others, NGS has led to the identification of disease-causing variants and novel drug targets and an improved understanding of complex biological events, e.g., the heterogeneity of tumors. 2022) recommend matching interventions to specific patient characteristics should be implemented early in the disease course. Fountzilas et al.

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The Pharma Data

DECEMBER 16, 2020

The Company will initiate its trial during the first quarter of 2021 to investigate the efficacy of Berubicin in adults with GBM who have failed first-line therapy. and 2 trials planned by our sublicensee WPD in Poland. . and 2 trials planned by our sublicensee WPD in Poland. About CNS Pharmaceuticals, Inc.

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The Pharma Data

APRIL 19, 2022

The site will produce Kite’s FDA approved CAR T-cell therapy used to treat blood cancer. The company is committed to advancing innovative medicines to prevent and treat life-threatening diseases, including HIV, viral hepatitis and cancer. Gilead Sciences, Inc. Source link: [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

JANUARY 15, 2021

15, 2021 — An inhaled medication might make every day physical activity a bit easier for patients with serious scarring of the lungs, a new clinical trial finds. 13 in the New England Journal of Medicine , involved patients with high blood pressure in the lungs caused by interstitial lung disease (ILD). FRIDAY, Jan.

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The Premier Consulting Blog

OCTOBER 11, 2022

Clinical trials for ultra-rare diseases can be particularly challenging to mount due to small, geographically-dispersed patient populations. For such trials, the US Food and Drug Administration (FDA) may allow the use of credible real-world data (RWD) and real-world evidence (RWE) in lieu of data collected in a Phase 3 trial.

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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

DECEMBER 3, 2020

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. The approval was based on data from the Phase 3 APeX-2 trial, which showed that ORLADEYO could significantly reduce attacks at the 24-week mark.

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