New Drug Approvals

DECEMBER 16, 2023

3 Additionally, iptacopan has the benefit of targeting factor B, which only affect the alternative complement pathway, leaving the classic and lectin pathway untouched for the body to still mount adequate immune responses against pathogens. of patients experienced a sustained hemoglobin improvement without transfusions respectively.

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The Pharma Data

DECEMBER 17, 2020

In the initial 275 patients, rates of adverse events (AEs) were similar among groups. Authorized Emergency Use Casirivimab and imdevimab injection is an investigational combination therapy and has been authorized by FDA for the emergency use described above. Casirivimab and imdevimab injection is not FDA approved for any use.

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Hospitals Production Clinical Trials Virus 52

DrugBank

APRIL 24, 2025

Azacitidine and decitabine , two FDA-approved DNMTi, are widely used for myelodysplastic syndromes and acute myeloid leukemia. In addition to supporting target identification and combination therapy development, DrugBank offers real-world data on adverse events and pharmacokinetics.

DNA 52

DNA Drugs Therapies Regulations 52

Drug Target Review

DECEMBER 5, 2024

1 In 2017, the US Food and Drug Administration (FDA) approved the first AAV-based gene replacement therapy (Luxturna), for Leber congenital amaurosis type 2. 2 Since then, the FDA has approved four more AAV-based gene therapies—Zolgensma, Hemgenix, Elevidys and Rocktavian—for treating various diseases.

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Therapies Virus DNA Compliance 52

DrugBank

FEBRUARY 12, 2025

The National Institutes of Health (NIH) Clinical Collection , a library of FDA-approved drugs, is widely used for HTS in drug repurposing initiatives. This approach allows researchers to assess the effects of drugs on specific cell types, providing insights into their mechanisms of action and potential for adverse events.

Cell Based Assays 52

Cell Based Assays Drugs Pharmacokinetics Disease 52

The Pharma Data

JULY 6, 2021

Decreased levels of PGRN, a key regulator of immune response, lysosomal function, and neuronal survival in the brain, are genetically linked to many neurodegenerative disorders. There are currently no FDA-approved treatment options for FTD. About the Progranulin-Elevating Monoclonal Antibodies – AL001 and AL101.

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Disease Science Treatment Clinical Development 52

The Pharma Data

DECEMBER 11, 2020

The Phase 3 trial is designed as a 1:1 vaccine candidate to placebo, randomized, observer-blinded study to obtain safety, immune response, and efficacy data needed for regulatory review. Vaccination providers must report Adverse Events in accordance with the Fact Sheet to VAERS at [link] or by calling 1-800-822-7967.

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Vaccine Clinical Trials Trials Licensing 52

The Pharma Data

OCTOBER 28, 2020

“We continue to see the strongest effects in patients who are most at risk for poor outcomes due to high viral load, ineffective antibody immune response at baseline, or pre-existing risk factors. Serious adverse events were numerically more frequent with placebo than REGN-COV2 treatment (0.8% Yancopoulos , M.D.,

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The Pharma Data

DECEMBER 27, 2020

To Our Shareholders, As we reach the end of an eventful 2020, we are inspired by the many healthcare providers and biopharmaceutical companies that worked to combat the COVID-19 pandemic. Finally, we seek to generate compelling clinical data to support moving into late-stage clinical trials and eventual marketing authorizations.

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The Pharma Data

JUNE 15, 2025

Food and Drug Administration (FDA)-approved gene therapy for the treatment of Duchenne muscular dystrophy (DMD). Sarepta’s team is exploring adding sirolimus — a well-established immunosuppressive medication — to the regimen in a way that might enable a greater degree of control over the patient’s immune response.

Immune Response 40

Immune Response Regulations Therapies Disease 40

The Pharma Data

AUGUST 15, 2021

Food and Drug Administration (FDA) has approved TICOVAC (tick-borne encephalitis (TBE) vaccine) for active immunization to prevent TBE in individuals 1 year of age and older. 1 TICOVAC is the only FDA-approved vaccine to help protect U.S. Following today’s FDA approval, the U.S.

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FDA Approval Vaccine FDA Virus 52

The Pharma Data

JUNE 18, 2021

Food and Drug Administration (FDA) has approved PREVNAR 20 (Pneumococcal 20-valent Conjugate Vaccine) for the prevention of invasive disease and pneumonia caused by the 20 Streptococcus pneumoniae (pneumococcus) serotypes in the vaccine in adults ages 18 years and older. Following today’s FDA approval, the U.S.

Vaccine 52

Vaccine FDA Approval FDA Disease 52

PerkinElmer

NOVEMBER 9, 2022

These cells overexpress PD-L1, leading to inhibition of cytotoxic T-cells by binding to the PD-1 receptor on activated T-cells, and subsequent evasion of an immune response. To date, the only FDA approved immune checkpoint inhibitors targeting the PD-1/PD-L1 pathway are monoclonal antibodies (mAbs).

Small Molecule 52

Small Molecule Research Immune Response FDA Approval 52

The Pharma Data

NOVEMBER 20, 2020

The clinical evidence from Regeneron’s outpatient trial suggests that monoclonal antibodies such as casirivimab and imdevimab have the greatest benefit when given early after diagnosis and in patients who have not yet mounted their own immune response or who have high viral load.

Therapies 52

Therapies FDA Clinical Trials Hospitals 52

The Pharma Data

DECEMBER 18, 2020

After a person receives this vaccine, their body produces copies of the spike protein, which does not cause disease, but triggers the immune system to learn to react defensively, producing an immune response against SARS-CoV-2. Director of the FDA’s Center for Biologics Evaluation and Research.

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Vaccine FDA Clinical Trials FDA Approval 52

The Pharma Data

DECEMBER 10, 2020

The most common adverse events of BNT162b2 were transient, mild to moderate pain at the injection site, fatigue and headache, and these generally resolved within two days. Rates of serious adverse events were similar between vaccine and placebo groups (0.6% BNT162b2 (SARS-CoV-2 vaccine) FDA Approval History.

Vaccine 52

Vaccine Trials Clinical Trials Therapies 52

The Pharma Data

APRIL 11, 2021

Opdivo plus chemotherapy also demonstrated improvements in key secondary endpoints, including major pathological response (MPR). Grade 3–4 treatment-related adverse events were reported in 34% vs. 37% in the Opdivo plus chemotherapy vs. chemotherapy alone arms, respectively. About CheckMate -816. About Lung Cancer.

Trials 52

Trials Treatment Clinical Trials Immune Response 52

The Pharma Data

DECEMBER 29, 2020

The analysis was prospectively designed to focus on patients who had not yet mounted their own immune response to SARS-CoV-2 (i.e., In the overall trial population, the incidence of serious adverse events was 21% for high dose, 20% for low dose and 24% for placebo. futility analysis). . high dose, 0.9% low dose, 1.4%

Hospitals 52

Hospitals Trials Clinical Trials Production 52

The Pharma Data

NOVEMBER 21, 2020

The clinical evidence from Regeneron’s outpatient trial suggests that monoclonal antibodies such as REGEN-COV2 have the greatest benefit when given early after diagnosis and in patients who have not yet mounted their own immune response or who have high viral load. None of the SAEs were considered to be related to study drug.

FDA 40

FDA Clinical Trials Hospitals Production 40

The Pharma Data

JANUARY 29, 2021

A review of adverse events indicated that a single-dose of Janssen’s COVID-19 vaccine candidate was generally well-tolerated. Overall serious adverse events (SAEs) reported were higher in participants who received placebo as compared to the active vaccine candidate. These statements are based on current expectations of future events.

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Vaccine Trials Pharmaceutical Companies Clinical Trials 40

The Pharma Data

AUGUST 11, 2020

Opdivo is a programmed death-1 (PD-1) immune checkpoint inhibitor that is designed to uniquely harness the body’s own immune system to help restore anti-tumor immune response. By harnessing the body’s own immune system to fight cancer, Opdivo has become an important treatment option across multiple cancers.

Therapies 52

Therapies Trials Disease Treatment 52

The Pharma Data

AUGUST 11, 2020

Opdivo is a programmed death-1 (PD-1) immune checkpoint inhibitor that is designed to uniquely harness the body’s own immune system to help restore anti-tumor immune response. By harnessing the body’s own immune system to fight cancer, Opdivo has become an important treatment option across multiple cancers.

Treatment 52

Treatment Trials Therapies Clinical Trials 52

Codon

JULY 14, 2024

This is particularly beneficial for those who need chronic transfusions and are therefore at risk of developing an immune response to transfusion products from other people, or for those with religious beliefs, like Jehovah’s Witnesses, whose faith prohibits blood transfusions from another person.

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Production Clinical Trials Trials Therapies 143

The Pharma Data

APRIL 4, 2023

Immune-mediated adverse reactions, which may be severe or fatal, can occur in any organ system or tissue, can affect more than one body system simultaneously, and can occur at any time after starting treatment or after discontinuation of treatment. All patients with a recurrence of ALT ≥3 ULN subsequently recovered from the event.

Treatment 40

Treatment FDA Approval FDA Therapies 40

The Pharma Data

OCTOBER 26, 2020

In addition, the observed median PFS shows that the regimen can induce a sustained and durable response, which is also shown consistently by the induction of a specific immune response. Continued approval may be contingent upon verification and description of clinical benefit in confirmatory trials.

Vaccine 40

Vaccine Trials Treatment Virus 40

The Pharma Data

APRIL 27, 2021

Sanofi expects 2021 business EPS ( 1) to grow high single digit (2) at CER, barring unforeseen major adverse events. At the end of March, the FDA approved Sarclisa ® in combination with carfilzomib and dexamethasone for patients with relapsed multiple myeloma. Sarclisa ® is already approved in the U.S Rare Blood Disorder.

Vaccine 52

Vaccine Production FDA Disease 52

The Pharma Data

JULY 15, 2021

Antibody and T-cell immune responses strong and stable at eight months after immunization Demonstrated neutralizing antibody activity against the Delta variant (B.1.617.2) In addition, the T-cell responses are especially strong and stable over time, which is also potentially important for activity against these variants.”.

Immune Response 52

Immune Response Vaccine Clinical Trials FDA 52

The Pharma Data

JANUARY 4, 2021

Tiragolumab is the first anti-TIGIT molecule to be granted BTD from the FDA, and the designation is based on randomized data from the Phase II CITYSCAPE trial. CITYSCAPE provides the first evidence that targeting both immune inhibitory receptors, TIGIT and PD-L1, may enhance anti-tumor activity by potentially amplifying the immune response.

Therapies 52

Therapies FDA Immune Response Treatment 52

Broad Institute

NOVEMBER 6, 2024

Now, they’ve uncovered the immune basis of checkpoint myocarditis , a severe form of heart inflammation that afflicts one percent of patients on an ICI therapy and up to 2 percent with certain combination therapy. These are home-run, breakthrough therapies, and quite an amazing success story. How did you go about launching this effort?

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