Eye on FDA

MARCH 5, 2023

Note these numbers cover meetings about drug applications only, not including vaccines or cellular therapies. That said, beginning in 2020 meetings began skewing to more negative outcomes and 2022 was the first year where the number of negative outcomes outweighed the number of positive outcomes.

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FDA Vaccine Drugs Therapies 72

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. ADUHELM is indicated for the treatment of Alzheimer’s disease.

Treatment 52

Treatment Therapies Disease FDA Approval 52

Drug Target Review

SEPTEMBER 6, 2024

What trends are driving the increased use of in vivo delivery methods in gene therapy? Being a specialist lentiviral vector CDMO, at VIVEbiotech, this annual growth rate has been higher with a CAGR from 2020 to 2023 of almost a 50 percent. How are new therapeutic areas emerging as potential targets for in vivo gene therapy?

Therapies 59

Therapies Vaccine Marketing Disease 59

Chemical Biology and Drug Design

DECEMBER 30, 2024

In 2020, two selective RET inhibitors, selpercatinib and pralsetinib were approved by the US FDA. However, high treatment costs and clinically acquired resistance (e.g., G810C/S/R) become the new challenges for RET-based therapies.

FDA 100

FDA Therapies Treatment Research 100

The Pharma Data

MAY 27, 2022

In the pivotal clinical study CARTITUDE-1, 98 percent of patients with relapsed or refractory multiple myeloma responded to a one-time treatment with ciltacabtagene autoleucel and 80 percent of patients who responded experienced a stringent complete response . In December 2017, Janssen Biotech, Inc. to develop and commercialise cilta-cel.[1].

Therapies 52

Therapies Treatment Pharmaceutical Companies Nurses 52

Drug Target Review

NOVEMBER 7, 2023

Chemists at Stanford University discovered how one of the pathways leading to this protein “woodchipper” works furthering the understanding of new therapeutics for autoimmune diseases, age-related disorders, and treatment-resistant cancers. Enzyme replacement therapy is a frequent treatment for these disorders.

Therapies 59

Therapies Science Disease Treatment 59

Advarra

JUNE 13, 2023

Research in gene therapies and genetically engineered drugs and vaccines are growing exponentially, and will only continue to become more popular. The accelerating gene therapy market is expected to grow globally by 16.6% between 2020-2027.

Therapies 52

Therapies Research Clinical Trials Trials 52

The Pharma Data

JANUARY 12, 2021

Preliminary revenue for the fourth quarter of 2020 is expected to be approximately $40.6 Preliminary revenue for the fourth quarter of 2020 is expected to be approximately $40.6 Preliminary revenue for the full year 2020 is expected to be approximately $125.4 2020 was a transformational year for Vapotherm.

Therapies 52

Therapies Production Marketing Regulations 52

The Pharma Data

MARCH 9, 2022

Investigators at Cedars-Sinai have identified a potential new therapy for COVID-19: a biologic substance created by reengineered human skin cells. Although still in the early stages, the findings open the possibility of having a new therapy for COVID-19 patients.

Treatment 52

Treatment Virus Engineering Therapies 52

The Pharma Data

FEBRUARY 8, 2021

Janssen Announces Treatment with ERLEADA ® (apalutamide) Significantly Improved Overall Survival in Patients with Metastatic Castration-Sensitive Prostate Cancer. In addition, health-related quality of life (HRQoL), per total Functional Assessment of Cancer Therapy–Prostate (FACT-P), continued to be maintained with ERLEADA ®.

Treatment 52

Treatment Pharmaceutical Companies Therapies Pharmaceuticals 52

Eye on FDA

AUGUST 18, 2020

But it is not likely to surprise anyone that the first half of 2020 has caused not only an increase in communication from FDA, but a focus as well. Approvals – There were 54 press statements regarding approvals so far in 2020, comparing to 42 approvals announced in 2019.

FDA 61

FDA Production Drugs Therapies 61

Advarra

NOVEMBER 29, 2022

The field of cell and gene therapies (CGT) is constantly evolving, and there has been significant progress in this area of research. However, despite the promise of these therapies, the regulations governing them lag the science, which in turn hinders the clinical translation of these novel medicines.

Therapies 52

Therapies Clinical Trials FDA Production 52

Drug Target Review

OCTOBER 26, 2023

Genome engineering and gene therapies that manipulate DNA sequences in cells have driven a biotechnological revolution over the past decade. 1 Adeno-associated virus (AAV) vectors are the leading platform for in vivo gene delivery for the treatment of various human diseases. Nature 578 , 229-236 (2020). References Doudna JA.

Engineering 105

Engineering Virus Therapies Immune Response 105

Drug Target Review

OCTOBER 4, 2023

Antibody-drug conjugates (ADCs) have been a groundbreaking approach to cancer treatment with their ability to deliver cytotoxic drugs directly to diseased cells while sparing healthy tissues. To overcome these obstacles, combination therapy, which delivers multiple small molecules, has emerged as a potential solution.

Small Molecule 126

Small Molecule Drugs Immune Response Targeted Protein Degradation 126

The Pharma Data

APRIL 17, 2021

TECFIDERA was approved under the National Medical Products Administration priority review process evaluating therapies with urgent clinical needs. Biogen’s expansion in China now includes treatment options approved for relapsing MS and spinal muscular atrophy. We thank the NMPA for undertaking priority review to approve TECFIDERA. “We

Treatment 52

Treatment Clinical Trials Disease Trials 52

Drug Target Review

SEPTEMBER 22, 2023

Organoid technologies are becoming an invaluable solution for preclinical research, with the ability to augment the development of personalised medicine, drug discovery and gene therapies. Organoids for cell therapy and drug discovery. Elsevier; 2020. 2020 Oct 7;21(10):571–84. 2020 Jun 8;4(9):863–74. 95: 729–38

Clinical Research 116

Clinical Research Research Drug Development Molecular Biology 116

The Pharma Data

APRIL 15, 2022

Designed computationally and refined in the laboratory, the new protein therapies thwarted infection by interfering with the virus’ ability to enter cells. The top protein neutralized the virus with similar or greater potency than antibody treatments with Emergency Use Authorization status from the U.S. 2 omicron subvariant.

Treatment 52

Treatment Virus Laboratories Engineering 52

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. Could you elaborate on the potential market size for SRP-001 and how it fits into the broader landscape of pain therapies? South Rampart Pharma, Inc.

Treatment 59

Treatment Clinical Trials Therapies Molecular Biology 59

The Pharma Data

SEPTEMBER 11, 2021

Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for empagliflozin as an investigational treatment for adults with heart failure with preserved ejection fraction (HFpEF), Boehringer Ingelheim and Eli Lilly and Company (NYSE: LLY) announced. o Completion: 2020. o Number of patients: 3,730.

Therapies 52

Therapies Hospitals Clinical Trials Trials 52

The Pharma Data

JANUARY 15, 2021

for the treatment of adult patients with locally advanced or metastatic HER2 positive gastric or gastroesophageal junction (GEJ) adenocarcinoma who have received a prior trastuzumab-based regimen. .–(BUSINESS WIRE) January 15, 2021 — Daiichi Sankyo Company, Ltd. In the U.S., Sammons Cancer Center and the W.W. Caruth, Jr.

Treatment 52

Treatment Trials Disease Therapies 52

DS in Pharmatics

JUNE 10, 2023

On April 12, 2023, BeNeLuxA1 announced that negotiations failed to secure reimbursement for Libmeldy, a gene therapy developed by Orchard Therapeutics for the treatment of metachromatic leukodystrophy, on grounds of price. In the UK, an initial application to NICE was rejected as well back in 2020. This is a huge […]

Marketing 66

Marketing Therapies Treatment 66

The Pharma Data

JUNE 28, 2021

Food and Drug Administration (FDA) and a marketing authorization application (MAA) to the European Medicines Agency (EMA) seeking approval of the novel combination of the cancer treatments copanlisib and rituximab. submission is for the treatment of patients with relapsed indolent B-cell non-Hodgkin’s lymphoma (B-iNHL). Fields, M.D.,

Treatment 52

Treatment Therapies Trials Clinical Development 52

The Pharma Data

MARCH 25, 2021

this week announced that Japan’s Ministry of Health, Labour and Welfare (MHLW) approved Breyanzi (lisocabtagene maraleucel: liso-cel), a CD19-directed chimeric antigen receptor (CAR) T cell therapy for the treatment of patients with relapsed or refractory (R/R) large B-cell lymphoma 1 and R/R follicular lymphoma. and Celgene K.K.,

Therapies 52

Therapies Trials Treatment Disease 52

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.

Disease 52

Disease FDA Treatment Clinical Trials 52

The Pharma Data

SEPTEMBER 27, 2021

Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for empagliflozin as an investigational treatment for adults with coronary failure with preserved ejection fraction (HFpEF), Boehringer Ingelheim and Eli Lilly and Company (NYSE: LLY) announced. About the EMPEROR coronary failure studies10,11.

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Therapies Clinical Trials Hospitals Trials 52

The Pharma Data

MAY 4, 2021

Tecentriq monotherapy has been shown to improve overall survival in people with high PD-L1 expression, when compared to chemotherapy, and therefore represents a new treatment option for people living with this difficult-to-treat disease.”. Grade 3–4 treatment-related adverse events were reported in 12.9% months (HR=0.76, 95% CI: 0.54–1.09).

Treatment 52

Treatment Therapies Disease Protein Expression 52

The Pharma Data

APRIL 27, 2022

Based on DESTINY-Breast04 results where AstraZeneca and Daiichi Sankyo’s Enhertu demonstrated a significant improvement in both progression-free survival and overall survival Enhertu has now been granted five Breakthrough Therapy Designations, including three in breast cancer and one in both lung and gastric cancers.

Therapies 52

Therapies Trials Protein Expression Treatment 52

DrugBank

AUGUST 9, 2024

However, searching for broader applicability and personalized approaches continues, as not all patients respond to these treatments. These new therapies have demonstrated significant LDL cholesterol reductions, but their high cost remains a barrier to widespread adoption.

Pharmaceuticals 76

Pharmaceuticals Research Pharmaceutical Companies Clinical Trials 76

The Pharma Data

MARCH 26, 2021

If approved, Tecentriq could offer people with a specific type of lung cancer a chemotherapy-free option in the first-line treatment setting. We remain committed to providing effective and tailored lung cancer treatment options, and this announcement is an important step toward this goal.”. months; hazard ratio [HR]=0.59, 95% CI: 0.40–0.89;

Treatment 52

Treatment Therapies Protein Expression Immune Response 52

The Pharma Data

FEBRUARY 22, 2022

Food and Drug Administration (FDA) for the adjunctive treatment of major depressive disorder (MDD) in patients who are receiving ongoing antidepressant therapy. In 2020, an estimated 21 million adults had at least one major depressive episode. VRAYLAR is also approved for the treatment of schizophrenia in adults (1.5

Treatment 52

Treatment FDA Clinical Trials FDA Approval 52

The Pharma Data

APRIL 27, 2022

ESCC is the most common type of esophageal cancer globally, with an estimated 604,000 new cases and 544,000 deaths from esophageal cancer internationally in 2020. As a leading global medicines company, we use innovative science and digital technologies to create transformative treatments in areas of great medical need.

Treatment 52

Treatment Clinical Development Therapies Clinical Trials 52

The Pharma Data

JULY 21, 2021

A BTD is intended to expedite the development and review of medications to treat a serious medical condition and is granted when preliminary clinical evidence indicates the investigational therapy may demonstrate substantial improvement over existing therapies. PRNewswire/ — AbbVie (NYSE: ABBV) announced today that the U.S.

Therapies 52

Therapies FDA Treatment Vaccine 52

The Pharma Data

FEBRUARY 4, 2021

3, 2021 /PRNewswire/ — AbbVie (NYSE: ABBV) announced financial results for the fourth quarter and full year ended December 31, 2020. AbbVie hosted an Immunology Strategic Update event on December 14, 2020 for members of the investment community. NORTH CHICAGO, Ill., Gonzalez , chairman and chief executive officer, AbbVie.

Production 52

Production Clinical Trials International Small Molecule 52

Creative Biolabs

MAY 25, 2022

Cancer has become the second largest health threat in the world, with about 10 million people dying of cancer in 2020. For decades, cytotoxic-based chemotherapy has been the main treatment for all kinds of cancers. These cytotoxic drugs include DNA base analogs, antimetabolic drugs, tubulin inhibitors, etc.

Therapies 52

Therapies Drugs DNA Treatment 52

Drug Target Review

JANUARY 15, 2024

1 Lung transplantation is the only viable treatment option, yet finding suitable lung donors is difficult. Pluripotent Stem Cell-Based Cell Therapy – Promise and Challenges. 2020 October 1 [2024 January 5]; 27(4):523-31. Annual Review of Pathology: Mechanisms of Disease. 2008 October 27 [2024 January 5]; 4:435-459.

Immune Response 113

Immune Response Disease Science Therapies 113

The Pharma Data

OCTOBER 8, 2021

Food and Drug Administration approved Rethymic for the treatment of pediatric patients with congenital athymia, a rare immune disorder. “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., Today, the U.S.

FDA Approval 52

FDA Approval FDA Treatment Disease 52

The Pharma Data

AUGUST 30, 2021

1 The benefit was independent of ejection fraction or diabetes status, 1 establishing empagliflozin as the first and only treatment to significantly improve outcomes for the full spectrum of heart failure patients. If approved, empagliflozin would become the first clinically proven therapy across the full heart failure spectrum.

Trials 52

Trials Therapies Hospitals Treatment 52