FDA and Patients - Pharma Rep Focus

Patient Dies in Gene Therapy Trial, But FDA Permits Neurogene to Proceed With Low Dose

MedCity News

NOVEMBER 22, 2024

A patient death in a clinical trial usually prompts the FDA to impose a clinical hold. But Neurogene is able to avoid a lengthy clinical trial pause largely because of its participation in an FDA pilot program intended to speed up the development of therapies for rare diseases.

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Astellas nabs class-first FDA approval for CLDN18.2 gastric cancer med Vyloy

Fierce Pharma

OCTOBER 18, 2024

After a prior rejection, Astellas can head into the weekend celebrating a class-first FDA approval for its new gastric cancer med Vyloy. Astellas' Vyloy won an FDA approval to treat gastric or gastroesophageal junction adenocarcinoma patients whose tumors are CLDN18.2

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'It was a good lesson': FDA reconsiders CAR-T boxed warning on secondary cancers, Peter Marks says

Fierce Pharma

NOVEMBER 1, 2024

An FDA announcement last year of an investigation into the risk of patients developing secondary cancers following the treatment of CAR-T cell therapies sent shock waves across the industry. | said in an interview with Fierce Pharma.

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Vertex Pharma’s Opioid Alternative Wins FDA Approval, First in a New Class of Pain Meds

MedCity News

JANUARY 31, 2025

Vertex Pharmaceuticals Jounavx is now FDA approved, providing patients a non-opioid option for treating acute pain. The post Vertex Pharmas Opioid Alternative Wins FDA Approval, First in a New Class of Pain Meds appeared first on MedCity News.

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What the FDA's New Dosage Guidance Means for the Future of Clinical Research

Speaker: Dr. Ben Locwin - Biopharmaceutical Executive & Healthcare Futurist

A recent draft from the FDA provides valuable insight. Learning Objectives: Dose-response curves and patient treatment: How do pharmacologic and toxicologic principles apply to the dosing of drugs in clinical development? What will the future hold for clinical research?

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FDA Clears Withings’ New Blood Pressure Monitor That Allows Providers to Communicate Directly with Patients

MedCity News

DECEMBER 17, 2024

This week, the FDA granted clearance to Withings BPM Pro 2 cellular blood pressure monitor. The device seeks to help providers scale remote patient monitoring for heart failure patients.

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FDA Patients

FDA considers 'regulatory action' for bluebird's Skysona after blood cancer reports

Fierce Pharma

DECEMBER 2, 2024

The FDA is investigating reports of patients develo | The FDA is investigating reports of patients developing blood cancer after receiving bluebird bio’s gene therapy Skysona and is evaluating the need for “further regulatory action,” the U.S. agency said last week.

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Rare Metabolic Disease That Leads to Childhood Obesity Gets Its First FDA-Approved Drug

MedCity News

MARCH 27, 2025

Soleno Therapeutics Vykat XR is now FDA approved for treating hyperphagia, or excessive hunger, in patients with Prader-Willi syndrome. The post Rare Metabolic Disease That Leads to Childhood Obesity Gets Its First FDA-Approved Drug appeared first on MedCity News.

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Look out, Pfizer: BridgeBio scores 'best-case' FDA nod for Attruby in cardiomyopathy

Fierce Pharma

NOVEMBER 25, 2024

With the nod, BridgeBio will challenge Pfizer’s powerhouse Vyndaqel franchise, which dominates the indication.

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FDA Approves Immunotherapy That Can Spare Bladder Cancer Patients From Radical Surgery

MedCity News

APRIL 23, 2024

ImmunityBio therapy Anktiva is now FDA approved for treating patients with non-muscle invasive bladder cancer. The post FDA Approves Immunotherapy That Can Spare Bladder Cancer Patients From Radical Surgery appeared first on MedCity News.

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Supernus Wearable Device for Dosing Parkinson’s Drug Lands Long-Awaited FDA Approval

MedCity News

FEBRUARY 4, 2025

Supernus Pharmaceuticals Onapgo provides a continuous infusion of apomorphine to manage off periods experienced by Parkinsons disease patients. Approval of Onapgo comes after the FDA twice turned back applications for the drug/device combination product.

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To Give Rare Disease Patients Hope, the FDA Must Re-Focus its Efforts

MedCity News

JULY 25, 2024

To bring cross-agency clinical leaders together, the FDA should establish a Center of Excellence for Rare Diseases. The post To Give Rare Disease Patients Hope, the FDA Must Re-Focus its Efforts appeared first on MedCity News.

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Cell Therapy Now Set to Reach More Patients With Expanded FDA Nod for 2 Multiple Myeloma Drugs

MedCity News

APRIL 8, 2024

The expanded FDA approvals makes these cell therapies more accessible to more patients, broadening the market for both products. The post Cell Therapy Now Set to Reach More Patients With Expanded FDA Nod for 2 Multiple Myeloma Drugs appeared first on MedCity News.

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FDA Approves First Drug for Treating Agitation in Alzheimer’s Patients

MedCity News

MAY 11, 2023

Rexulti, a drug for schizophrenia and depression, now has an additional approval for treating agitation in Alzheimer’s disease patients. The FDA decision makes the Otsuka Pharmaceutical and Lundbeck product the first drug approved for this indication.

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FDA warns patients about compounded versions of Novo Nordisk's Ozempic, Wegovy

Fierce Pharma

MAY 31, 2023

FDA warns patients about compounded versions of Novo Nordisk's Ozempic, Wegovy kdunleavy Wed, 05/31/2023 - 13:02

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GSK Multiple Myeloma Drug’s DREAMM Comeback Continues With Phase 3 Data at ASH

MedCity News

DECEMBER 9, 2024

Results from the Phase 3 DREAMM-7 study show GSK drug Blenrep is helping multiple myeloma patients live longer compared to treatment with blockbuster Johnson & Johnson drug Darzalex. The post GSK Multiple Myeloma Drugs DREAMM Comeback Continues With Phase 3 Data at ASH appeared first on MedCity News.

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FDA approval of AstraZeneca drug is first for a new group of breast cancer patients

MedCity News

AUGUST 5, 2022

Enhertu, a breast cancer drug from AstraZeneca and Daiichi Sankyo, is now FDA approved for treating patients whose tumors express low levels of the protein HER2. The decision makes the infused therapy the first targeted treatment for this new category of breast cancer patients.

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Patient Death Prompts FDA Hold on Tests of Drug in Atopic Dermatitis & Asthma

MedCity News

FEBRUARY 20, 2024

The FDA clinical hold follows a patient death from liver failure that may be associated with zelnecirnon, an experimental Rapt Therapeutics drug. The biotech notes that no other patient has experienced liver problems and this safety signal has not been seen in any other tests of the molecule.

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The FDA Is Opening the Use of Registry Data in Drug Development – What Does It Mean for Patient Advocacy Groups?

MedCity News

DECEMBER 2, 2022

FDA’s draft guidances on real-world data do not provide strict requirements for industry stakeholders to abide by, but they offer patient advocacy organizations with a clearer understanding of how to model their registries to have a more substantial impact for their patient communities.

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Sanofi’s FDA Nod in Hemophilia Gives Patients More Convenience, Brings Roche New Competition

MedCity News

FEBRUARY 23, 2023

FDA approval of Sanofi hemophilia A drug Altuviiio provides patients with a therapeutic option that lasts longer than most other products available, including one already marketed by the French pharma giant. Altuviiio also gives Sanofi a way to better compete against blockbuster Roche drug Hemlibra.

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Eli Lilly Alzheimer’s Drug Lands FDA Approval, Introducing Competition to Eisai’s Leqembi

MedCity News

JULY 2, 2024

Eli Lilly’s Kisunla is now FDA approved for treating patients in the early stages of Alzheimer’s disease. In addition to slowing cognitive decline and disease progression, clinical trial results showed that amyloid beta plaques were reduced to the point where some patients could stop taking the infused therapy.

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FDA investigates 'serious risk' of secondary cancer following CAR-T treatment

Fierce Pharma

NOVEMBER 28, 2023

In a blow to CAR-T therapies, the FDA is investigating a “serious risk” of patients developing new cancers after treatment with these highly efficacious oncology drugs. |

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Full FDA Approval of Travere’s Kidney Drug Stiffens Competition With Novartis, Calliditas

MedCity News

SEPTEMBER 6, 2024

The FDA decision for Travere Therapeutics’ Filspari also expands its addressable patient population. The post Full FDA Approval of Travere’s Kidney Drug Stiffens Competition With Novartis, Calliditas appeared first on MedCity News.

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FDA Says ‘No’ to Novo Nordisk’s Once-Weekly Insulin

MedCity News

JULY 12, 2024

The FDA rejected Novo Nordisk’s biologics license application for icodec, citing questions about the manufacturing process for this once weekly insulin as well as its use by type 1 diabetes patients. But there are several high-profile FDA approvals in our recap of recent regulatory news.

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Ascendis Pharma Lands Long-Awaited FDA Drug Approval in Rare Hormone Deficiency

MedCity News

AUGUST 12, 2024

FDA approval of Ascendis Pharma’s Yorvipath is just in time for patients with hypoparathyroidism as Takeda Pharmaceutical will soon stop making the only drug therapy for the rare disorder. The post Ascendis Pharma Lands Long-Awaited FDA Drug Approval in Rare Hormone Deficiency appeared first on MedCity News.

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FDA Drug Approval Marks a New Day for Treating Pediatric Brain Cancer

MedCity News

APRIL 24, 2024

Day One Biopharmaceuticals drug Ojemda is now FDA-approved for advanced pediatric low-grade glioma, the most common type of brain cancer in children. The regulatory decision for Ojemda covers a broader swath of patients than a drug combination from Novartis approved for treating this childhood cancer.

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An Attack on Patients’ Right to Informed Consent

MedCity News

MARCH 6, 2024

This case, about the claim that Merck failed to warn patients about the risk of typical femoral fracture, reaches past this suit to potentially change pharmaceutical companies’ relationship with the FDA.

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FDA Rejection Delays Regeneron From Competing With J&J, Pfizer in Multiple Myeloma

MedCity News

AUGUST 21, 2024

Regeneron Pharmaceuticals said the FDA cited no approvability concerns for its multiple myeloma drug, linvoseltamab, other than previously identified issues with a contract manufacturer. The post FDA Rejection Delays Regeneron From Competing With J&J, Pfizer in Multiple Myeloma appeared first on MedCity News.

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J&J angles Darzalex for first move into early-stage myeloma with trial win, FDA filing

Fierce Pharma

NOVEMBER 8, 2024

After a positive phase 3 trial, Johnson & Johnson hopes its Darzalex could become the first treatment option for patients with smoldering multiple myeloma, an early-stage form of the blood diso | After a positive phase 3 trial, Johnson & Johnson hopes its Darzalex could become the first treatment option for patients with smoldering multiple (..)

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FDA Nod in Duchenne Helps Wider Swath of Patients With the Rare Muscle Disease

MedCity News

MARCH 22, 2024

FDA approval of Italfarmaco’s Duvyzat is the first nonsteroidal drug to pass the agency’s regulatory bar for treating this rare disease. Corticosteroids are a standard first-line treatment for Duchenne muscular dystrophy, but these drugs have many side effects.

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FDA Forms New Digital Health Advisory Committee to Cover Growing Role of Tech

MedCity News

OCTOBER 15, 2023

The FDA’s digital health advisory committee will discuss technologies such as artificial intelligence and machine learning, virtual reality, and digital therapeutics as well as topics like decentralized clinical trials and patient-generated health data. Committee member nominations are due in December.

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FDA Probes New Cases of Cancer That May Stem From Cancer Cell Therapies

MedCity News

NOVEMBER 28, 2023

CAR T-therapies for cancer are now under tighter FDA scrutiny as the agency opens an inquiry regarding reports that some patients who received these cell therapies went on to develop new cases of cancer. The FDA said benefits of these therapies still outweigh their risks, but it is evaluating the need for regulatory action.

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FDA Mulls Policy Switch on Switching Study Requirement for Biosimilars

MedCity News

JUNE 20, 2024

FDA draft guidance eliminates the requirement that biosimilars produce clinical data to show they are interchangeable with their reference drugs. Some lawmakers have said these so-called switching studies add time and expense to the development of biosimilars, delaying them from reaching patients.

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J&J multiple myeloma drug gets FDA nod, bringing patients an easier dosing option

MedCity News

OCTOBER 26, 2022

FDA approval of Tecvayli, from Johnson & Johnson subsidiary Janssen Biotech, makes it the newest therapy that treats multiple myeloma by targeting the cancer protein BCMA.

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Heartburn Drug from Phathom Pharma Expands Its Approval to Cover More GERD Patients

MedCity News

JULY 18, 2024

Expanded FDA approval of Phathom Pharmaceuticals drug Voquezna now includes patients with non-erosive gastroesophageal reflux disease (GERD). The post Heartburn Drug from Phathom Pharma Expands Its Approval to Cover More GERD Patients appeared first on MedCity News.

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Novo Nordisk’s Diabetes Pill Rybelsus Gets FDA O.K. as First-Line Therapy

MedCity News

JANUARY 13, 2023

The FDA approved a change in Rybelsus’s label, allowing the drug to be used as an initial therapy for type 2 diabetes patients. The once-daily pill has already become a blockbuster product for Novo Nordisk.

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Pfizer Meningococcal Vaccine Nabs FDA Nod, Offering More Convenient Dosing

MedCity News

OCTOBER 22, 2023

The FDA approved Pfizer’s Penbraya for vaccinating against five bacterial groups that can cause meningococcal disease. Pfizer already has vaccines covering those groups, but Penbraya addresses all of them in a single vaccine, making dosing easier for patients.

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GSK Gets FDA Nod for Drug Treating Anemia From Chronic Kidney Disease

MedCity News

FEBRUARY 2, 2023

The FDA approved GSK drug Jesduvroq for treating anemia in patients with chronic kidney disease. The once-daily pill is the first oral medicine to pass FDA muster in this indication, giving patients a more convenient alternative to injectable anemia therapies.

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Medtronic Gains FDA Approval for Newest Insulin Pump

MedCity News

APRIL 25, 2023

Medtronic recently received FDA approval for its MiniMed 780G system, which is an insulin pump that automatically adjusts and corrects type 1 diabetes patients’ glucose levels every five minutes.

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Provention Wins FDA Approval for First Drug to Delay Type 1 Diabetes Progression

MedCity News

NOVEMBER 18, 2022

Type 1 diabetes patients now have a new therapeutic option for the autoimmune disorder. Provention Bio drug Tzeild has received FDA approval for delaying the onset of the most severe stage of this chronic disease.

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GrayMatters Health Earns FDA Clearance for PTSD Neuromodulation Device

MedCity News

MARCH 23, 2023

GrayMatters Health recently received 510(k) clearance from the FDA to market its flagship product, which is a self-neuromodulation digital therapy for PTSD. The device uses neurofeedback on specific biomarkers to help patients identify and utilize their own mental strategy for lowering their emotional response.

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Verona Pharma Drug With Dual Mechanism Lands FDA Approval in COPD

MedCity News

JUNE 27, 2024

Verona Pharma’s COPD drug Ohtuvayre is a single small molecule that blocks two targets to bring patients both bronchodilation and anti-inflammatory effects. The post Verona Pharma Drug With Dual Mechanism Lands FDA Approval in COPD appeared first on MedCity News.

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BioLineRx's Aphexda scores FDA nod to help myeloma patients undergo stem cell transplants

Fierce Pharma

SEPTEMBER 11, 2023

After 10 years of stagnation, a new FDA approval could help more cancer patients become eligible for stem cell transplants. |

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FDA Patients

FDA issues first recommendations on AI for drug development

European Pharmaceutical Review

JANUARY 7, 2025

The US Food and Drug Administration (FDA) has for the first time, issued draft guidance on using artificial intelligence (AI) intended to support a regulatory decision about a drug or biological product. FDA has previously published a discussion paper that was produced to inform its guidance on AI in pharmaceutical drug development.

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Patient Dies in Gene Therapy Trial, But FDA Permits Neurogene to Proceed With Low Dose

Astellas nabs class-first FDA approval for CLDN18.2 gastric cancer med Vyloy

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'It was a good lesson': FDA reconsiders CAR-T boxed warning on secondary cancers, Peter Marks says

Webinars

Vertex Pharma’s Opioid Alternative Wins FDA Approval, First in a New Class of Pain Meds

What the FDA's New Dosage Guidance Means for the Future of Clinical Research

FDA Clears Withings’ New Blood Pressure Monitor That Allows Providers to Communicate Directly with Patients

FDA considers 'regulatory action' for bluebird's Skysona after blood cancer reports

Rare Metabolic Disease That Leads to Childhood Obesity Gets Its First FDA-Approved Drug

Look out, Pfizer: BridgeBio scores 'best-case' FDA nod for Attruby in cardiomyopathy

FDA Approves Immunotherapy That Can Spare Bladder Cancer Patients From Radical Surgery

Supernus Wearable Device for Dosing Parkinson’s Drug Lands Long-Awaited FDA Approval

To Give Rare Disease Patients Hope, the FDA Must Re-Focus its Efforts

Cell Therapy Now Set to Reach More Patients With Expanded FDA Nod for 2 Multiple Myeloma Drugs

FDA Approves First Drug for Treating Agitation in Alzheimer’s Patients

FDA warns patients about compounded versions of Novo Nordisk's Ozempic, Wegovy

GSK Multiple Myeloma Drug’s DREAMM Comeback Continues With Phase 3 Data at ASH

FDA approval of AstraZeneca drug is first for a new group of breast cancer patients

Patient Death Prompts FDA Hold on Tests of Drug in Atopic Dermatitis & Asthma

The FDA Is Opening the Use of Registry Data in Drug Development – What Does It Mean for Patient Advocacy Groups?

Sanofi’s FDA Nod in Hemophilia Gives Patients More Convenience, Brings Roche New Competition

Eli Lilly Alzheimer’s Drug Lands FDA Approval, Introducing Competition to Eisai’s Leqembi

FDA investigates 'serious risk' of secondary cancer following CAR-T treatment

Full FDA Approval of Travere’s Kidney Drug Stiffens Competition With Novartis, Calliditas

FDA Says ‘No’ to Novo Nordisk’s Once-Weekly Insulin

Ascendis Pharma Lands Long-Awaited FDA Drug Approval in Rare Hormone Deficiency

FDA Drug Approval Marks a New Day for Treating Pediatric Brain Cancer

An Attack on Patients’ Right to Informed Consent

FDA Rejection Delays Regeneron From Competing With J&J, Pfizer in Multiple Myeloma

J&J angles Darzalex for first move into early-stage myeloma with trial win, FDA filing

FDA Nod in Duchenne Helps Wider Swath of Patients With the Rare Muscle Disease

FDA Forms New Digital Health Advisory Committee to Cover Growing Role of Tech

FDA Probes New Cases of Cancer That May Stem From Cancer Cell Therapies

FDA Mulls Policy Switch on Switching Study Requirement for Biosimilars

J&J multiple myeloma drug gets FDA nod, bringing patients an easier dosing option

Heartburn Drug from Phathom Pharma Expands Its Approval to Cover More GERD Patients

Novo Nordisk’s Diabetes Pill Rybelsus Gets FDA O.K. as First-Line Therapy

Pfizer Meningococcal Vaccine Nabs FDA Nod, Offering More Convenient Dosing

GSK Gets FDA Nod for Drug Treating Anemia From Chronic Kidney Disease

Medtronic Gains FDA Approval for Newest Insulin Pump

Provention Wins FDA Approval for First Drug to Delay Type 1 Diabetes Progression

GrayMatters Health Earns FDA Clearance for PTSD Neuromodulation Device

Verona Pharma Drug With Dual Mechanism Lands FDA Approval in COPD

BioLineRx's Aphexda scores FDA nod to help myeloma patients undergo stem cell transplants

FDA issues first recommendations on AI for drug development

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