FDA and Leads - Pharma Rep Focus

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 once-daily pill is the first approved therapy for this rare disease, a leading cause of childhood obesity.

Leads

Leads FDA Patients Biopharma

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

MedCity News

DECEMBER 9, 2024

The data, presented Monday during the annual meeting of the American Society of Hematology, are part of a submission now under FDA review that could lead to Blenreps return to the market. The post GSK Multiple Myeloma Drugs DREAMM Comeback Continues With Phase 3 Data at ASH appeared first on MedCity News.

FDA

FDA Leads Marketing Patients

Novo Nordisk Commits $285M to Ascendis to Kick Off Alliance on Longer-Acting GLP-1 Drugs

MedCity News

NOVEMBER 4, 2024

The Novo Nordisk/Ascendis partnership’s lead program is once-monthly injectable semaglutide for type 2 diabetes and obesity. This product candidate will be developed with Ascendis Pharma technology that has already yielded two FDA-approved products with longer dosing intervals.

Pharma

Pharma FDA Leads Biopharma

FDA Reconsideration Leads to Approval of Takeda Drug for Rare Esophagus Disorder

MedCity News

FEBRUARY 12, 2024

FDA approval of Takeda Pharmaceutical drug Eohilia introduces a new therapeutic option for patients with eosinophilic esophagitis, inflammation of the esophagus that causes swallowing difficulty. It will compete against Dupixent, a Sanofi and Regeneron Pharmaceuticals drug already approved for this disorder.

FDA

FDA Leads Pharmaceutical Patients

FDA setback leads women’s health biotech ObsEva to give up on uterine fibroids drug

MedCity News

JULY 27, 2022

ObsEva’s uterine fibroids drug linzagolix has hit a snag at the FDA, portending a likely delayed regulatory decision. Rather than stick it out and see if its small molecule can match up against commercialized products from AbbVie and Myovant, ObsEva plans to give up rights to the drug as part of a corporate restructuring.

FDA

FDA Leads Biopharma Pharma

New Hemophilia Data Lead FDA to Delay Decision for BioMarin Gene Therapy

MedCity News

MARCH 7, 2023

BioMarin Pharmaceutical’s submission of additional data for patients treated with its hemophilia A gene therapy, Roctavian, mean that the FDA will push out a regulatory decision to early summer. But it could be worth the wait as analysts anticipate FDA approval and project Roctavian becoming one of BioMarin’s biggest products.

FDA

FDA Leads Pharmaceutical Patients

After Data Miss in Lead Cancer Indication, Replimune Maps Path for Oncolytic Virus

MedCity News

DECEMBER 5, 2023

Replimune’s lead therapeutic candidate did not meet the two main goals of a study intended to support an FDA submission in cutaneous squamous cell carcinoma. But Replimune’s therapy, an oncolytic virus, has better data in melanoma, and the company plans a 2024 FDA submission in this skin cancer.

Leads

Leads FDA Biopharma Pharma

Cardiac Complication Leads FDA to Place Foghorn Cancer Drug Under Partial Hold

MedCity News

APRIL 24, 2023

A clinical trial for a Foghorn Therapeutics cancer drug is under a partial FDA hold after a patient in the early-stage study developed a cardiovascular complication. It’s the biotech’s second partial hold in the past year.

FDA

FDA Leads Patients Biopharma

Preclinical Safety Signal Prompts FDA Clinical Hold on Neumora Schizophrenia Drug

MedCity News

APRIL 15, 2024

The FDA clinical hold keeps Neumora Therapeutics from catching up to the field of biopharmaceutical companies pursuing the same target for schizophrenia. But Neumora remains on track with its lead program, a different drug in late-stage clinical testing for major depressive disorder.

Safety

Safety FDA Leads Biopharma

Vertex’s CRISPR Gene Therapy Lands Another FDA Nod in a Rare Blood Disease

MedCity News

JANUARY 16, 2024

The FDA approved Vertex Pharmaceuticals’ gene therapy Casgevy for treating beta thalassemia, an inherited blood disease that leads to low levels of functioning hemoglobin. Last month, the one-time treatment won its first FDA nod for treating sickle cell disease.

FDA

FDA Pharmaceutical Leads Biopharma

Liver injury leads FDA to pause Sanofi’s tests of MS drug acquired in $3.7B deal

MedCity News

JUNE 30, 2022

The FDA placed a partial clinical hold on late-stage tests of Sanofi multiple sclerosis drug tolebrutinib after some patients developed drug-induced liver injury. tests are paused, Sanofi said clinical testing in other countries is continuing with additional safety monitoring.

FDA

FDA Leads Safety Patients

Amylyx ALS drug’s winding regulatory path leads to long-awaited FDA approval

MedCity News

SEPTEMBER 30, 2022

An Amylyx Pharmaceuticals drug developed to slow the progression of amyotrophic lateral sclerosis is now approved, making it just the third FDA-approved treatment for the disease. Amylyx will market its new product under the name “Relyvrio.”.

FDA

FDA Leads Pharmaceutical Marketing

In gene therapy race, bluebird leads Vertex in activating treatment centers but trails in patient starts

Fierce Pharma

MAY 9, 2024

While Vertex leads in patient starts, bluebird has activated more treatment centers. It’s not an easy question to answer.

Leads

Leads Patients FDA Pharmaceutical

FDA Approves Bausch + Lomb Drug With a New Approach to Dry Eye Disease

MedCity News

MAY 19, 2023

Bausch + Lomb drug Miebo is now FDA approved as a new treatment for dry eye disease. Unlike many products that rewet the eye, Miebo is designed to address one of the factors that leads to dry eyes.

FDA

FDA Leads Biopharma Pharma

FDA wants classwide boxed warning on all commercial CAR-T therapies amid secondary cancer safety probe

Fierce Pharma

JANUARY 22, 2024

As many have feared, the FDA’s investigation into secondary T-cell cancers following treatment with existing CAR-T therapies is poised to lead to a classwide black box warning. |

FDA

FDA Safety Leads

With FDA approval, Eli Lilly's weight-loss drug Zepbound arrives to challenge Novo Nordisk

Fierce Pharma

NOVEMBER 8, 2023

In the Big Pharma battle to treat obesity, Novo Nordisk sprinted to an early lead, but Eli Lilly is catching up quickly. | In the Big Pharma battle to treat obesity, Novo Nordisk sprinted to an early lead, but Eli Lilly is catching up quickly.

FDA

FDA Pharma Leads

Safety Risks and FDA Hold Lead Gilead to Stop Work on Drug in Blood Cancers

MedCity News

FEBRUARY 7, 2024

Gilead Sciences said an independent review of interim Phase 3 data found the immunotherapy, magrolimab, led to a higher risk of death. Though Gilead is discontinuing further development of the drug in blood cancers, a review is ongoing in solid tumors.

Safety

Safety Leads FDA Biopharma

BMS, Mirati's Krazati cements lead over Amgen's Lumakras with FDA approval in colorectal cancer

Fierce Pharma

JUNE 24, 2024

Despite coming to market second, Bristol Myers Squibb’s Mirati Therapeutics-inherited cancer med Krazati has gained the lead on its Amgen rival Lumakras in a heated KRAS race. Late Friday, the U.S.

Leads

Leads FDA Marketing

FDA Approval of Travere Kidney Disease Drug Sets Up Blockbuster Expectations

MedCity News

FEBRUARY 21, 2023

Travere Therapeutics drug Filspari won accelerated FDA approval to treat a rare disorder that can lead to kidney failure. The small molecule is also in late-stage testing for another rare kidney disease; analysts say each indication represents a blockbuster opportunity.

FDA

FDA Leads Biopharma Pharma

FDA approves first-in-class oral antibiotic

European Pharmaceutical Review

MARCH 26, 2025

The US Food and Drug Administration (FDA) has approved Blujepa ( gepotidacin ) antibiotic for uncomplicated urinary tract infections (uUTIs) in female adults and paediatric patients aged 12 years old and over. The post FDA approves first-in-class oral antibiotic appeared first on European Pharmaceutical Review.

Food and Drug Administration

Food and Drug Administration FDA Medicine Food

RFK Jr. could 'cost lives in this country' if he pursues changes to US vaccine policy, Gottlieb says

Fierce Pharma

DECEMBER 2, 2024

s appointment last month to lead the U.S. The former FDA Commissioner pointed to the threat of measles if vaccination rates were to decline by 5%. Kennedy Jr.’s Department of Health and Human Services immediately sent waves of speculation swirling throughout the biopharma industry. |

Biopharma

Biopharma FDA Leads

Fierce Pharma Asia—BeiGene, Novartis' TIGIT divorce; Takeda's FDA withdrawal; Eisai's new Alzheimer's lead

Fierce Pharma

JULY 13, 2023

Takeda pulled the FDA filing for its dengue vaccine after the FDA requested more data. BeiGene lost its TIGIT inhibitor collaboration with Novartis but signed an antibody-drug conjugate deal with Duality Biologics. Novartis walked away from BeiGene's TIGIT inhibitor. And more.

FDA

FDA Leads Pharma

Patient Death Leads to Pause in Test of 2seventy Bio Cell Therapy for Leukemia

MedCity News

JUNE 14, 2023

The FDA has been notified and an investigation is underway. 2seventy Bio reported that an acute myeloid leukemia patient treated with its experimental cell therapy has died, prompting research partner Seattle Children’s Research Institute to pause the Phase 1 study.

Leads

Leads Patients FDA Biopharma

By Eliminating Requirement for Animal Testing, FDA Modernization Act Allows Faster, More Cost-effective Drug Development

MedCity News

DECEMBER 26, 2022

By easing regulatory requirements for animal testing, the Act allows scientists to use innovative, leading-edge technologies more fully in future drug development strategies. .

FDA

FDA Leads Biopharma

New FDA head needs to examine the accelerated approval program

World of DTC Marketing

OCTOBER 21, 2021

The FDA’s “accelerated approval” program expedites the evaluation process for new treatments so that patients can have access to them sooner. Between 2011 and 2018, cumulative spending on 44 FDA-approved oral targeted therapy drugs was $3.5 appeared first on World of DTC Marketing.com.

FDA

FDA Insurance Prescription Medicine

AbbVie pays $255M to a buy biotech with drug candidate for fatal lung disorder

MedCity News

OCTOBER 21, 2022

based biotech’s lead program is an antibody drug in preclinical development for idiopathic pulmonary fibrosis, a chronic lung disorder with few FDA-approved treatments. AbbVie acquired DJS Antibodies for $255 million up front.

FDA

FDA Leads Biopharma Pharma

Amgen Osteoporosis Drug Prolia Is Hit With FDA’s Strictest Warning

MedCity News

JANUARY 21, 2024

The FDA said Amgen’s Prolia may lead to low levels of calcium, or hypocalcemia.

FDA

FDA Medicine Leads Biopharma

Apellis Drug Becomes First Approved Therapy for Vision-Loss Disorder Geographic Atrophy

MedCity News

FEBRUARY 17, 2023

The FDA approved Apellis Pharmaceuticals’ Syfovre as a treatment for geographic atrophy, a retinal disorder that is a leading cause of blindness. The drug, administered as an injection into the eye, will launch in March at the price of $2,190 per vial.

FDA

FDA Pharmaceutical Leads Biopharma

Eisai's US CEO and Alzheimer's lead Ivan Cheung telegraphs retirement days after Leqembi's full FDA nod

Fierce Pharma

JULY 11, 2023

FDA, Eisai’s global Alzheimer’s disease officer is getting ready to hang up his hat. With Cheung headed for the exit, Keisuke Naito—the 34-year-old son of Eisai’s CEO Haruo Naito—has taken up the lead Alzheimer’s mantle, the company said Tuesday. Right after Leqembi's full approval from the U.S.

Leads

Leads FDA

FDA’s ‘ivory tower thinking’ ignores promising biomarkers, says a rare disease CEO

PharmaVoice

OCTOBER 4, 2023

The FDA wouldn’t review a drug that was 90% effective. In rare diseases, this is too common, said the UltraGenyx CEO and a leading rare disease researcher.

FDA

FDA Leads

Otsuka Commits $800M in M&A Deal Bringing a Phase 3-Ready Metabolic Disorder Drug

MedCity News

AUGUST 1, 2024

Otsuka Pharmaceutical is acquiring Jnana Therapeutics, whose lead drug is a potential treatment for the rare metabolic disorder phenylketonuria (PKU). Jnana’s drug could provide an alternative to two FDA-approved PKU medicines from BioMarin Pharmaceutical.

Pharmaceutical

Pharmaceutical Medicine FDA Leads

With FDA filing on the horizon, Karuna appoints new CEO

PharmaVoice

DECEMBER 9, 2022

Allergan alum Bill Meury will take the helm as current CEO Steve Paul transitions to lead R&D.

FDA

FDA Leads

Regeneron hopes for quick FDA decision on high-dose Eylea as Roche's rival surges

Fierce Pharma

AUGUST 3, 2023

In a high-stakes race of their eye disease drugs, Roche has sprinted to a significant lead over Regeneron. Playing catch-up with Roche in a war of eye disease treatments, Regeneron hopes to gain an FDA decision for its recently rejected high-dose Eylea in this quarter.

FDA

FDA Leads

An audiologist’s take on the news that OTC hearing aids are on their way to consumers

MedCity News

AUGUST 21, 2022

If FDA’s decision on making hearing aids available over the counter leads to greater adoption, it could encourage consumers to learn more about their condition, and therefore improve the health literacy of the public.

FDA

FDA Leads Medical

Here’s Why Novartis Is Paying $500M to Acquire a Preclinical RNA Startup

MedCity News

JULY 18, 2023

Novartis’s acquisition of DTx Pharma brings a lead program for a rare disease with no FDA-approved therapies. But the deal could also help the pharma giant bring new RNA therapies to new places in the body, a top Novartis executive explained.

Pharma

Pharma FDA Leads Biopharma

Applied Tx craters as FDA rejects lead product candidate

pharmaphorum

NOVEMBER 28, 2024

Applied Therapeutics plummets after the FDA turns down its marketing application for govorestat as a treatment for galactosaemia

FDA

FDA Leads Marketing

GSK's Arexvy maintains its RSV market dominance, looks to key label expansion as Moderna's mRNA threat looms

Fierce Pharma

MAY 1, 2024

Even with the threat of a potential new competitor in the respiratory syncytial virus (RSV) vaccine race on the horizon, GSK and its leading Arexvy shot aren’t sweating. | Next up is a key FDA decision on the vaccine's use in the 50-to-59 age group, which would add another 50 million U.S. adults to its reach.

Marketing

Marketing FDA Leads

Pharma is big business whose goal is to maximize shareholder value

World of DTC Marketing

JULY 9, 2021

SUMMARY: Pfizer asks for accelerated approval for a COVID booster shot even though the FDA says it’s unnecessary. From 2016 to 2020, the 14 leading drug companies spent $577 billion on stock buybacks and dividends—$56 billion more than they spent on R&D over the same period.”

Pharma

Pharma FDA Leads Competition

A scientific approach to irrational consumer choices

World of DTC Marketing

DECEMBER 2, 2021

GOOD MORNING: The FDA is taking a scientific approach to evaluating direct-to-consumer (DTC) drug advertising, focusing on measuring consumer perceptions of the information presented in ads. ” So, are we supposed to believe that better images in DTC ads lead to patients asking for an Rx? ” Uh…no.

Food and Drug Administration

Food and Drug Administration Side effects FDA Prescription

Bluebird touts Zynteglo, Skysona launch progress as FDA decision on sickle cell therapy nears

Fierce Pharma

AUGUST 8, 2023

After closing shop in Europe two years ago, bluebird bio still aims to become an industry-leading gene therapy maker in the U.S. Bluebird aims to become an industry-leading gene therapy maker. First, it’s hoping new launches will help keep its doors open over the next 12 months.

Leads

Leads FDA

European authorities recognised for leading medicine regulatory oversight

European Pharmaceutical Review

MAY 22, 2024

The US Food and Drug Administration (FDA) Now, overall, the WLA listing comprises of 36 regulatory authorities from 34 Member States. The post European authorities recognised for leading medicine regulatory oversight appeared first on European Pharmaceutical Review.

Medicine

Medicine Leads Food and Drug Administration Safety

The debate on DTC marketing is going to heat up again

World of DTC Marketing

JULY 21, 2021

DTC ads do NOT lead to unnecessary Rxs. The FDA needs to study what people do when they see a DTC ad. 4our: DTC marketing should be illegal because it leads to unnecessary Rx’s. The FDA, in its wisdom, has never studied what patients actually do when they see a DTC TV ad. Again, this is a huge misunderstanding.

Marketing

Marketing Prescription Side effects Doctors

Medical Devices Are Evolving Toward Specialized Solutions

MedCity News

JULY 27, 2023

It is a minimally invasive treatment that has shown tremendous promise in addressing vascular calcification, which increases the risk of heart attacks, strokes, or blood clots, and can lead to limb amputations. In February of 2019, Johnson & Johnson acquired Auris Health and its FDA-cleared Monarch platform for $5.75

Specialization

Specialization Medical Medicine Leads

FDA grants fast track status to FELIQS’s FLQ-101 to prevent ROP

Pharmaceutical Technology

NOVEMBER 4, 2024

The US FDA has granted fast track designation to FELIQS’ lead product FLQ-101 for preventing retinopathy of prematurity (ROP).

FDA

FDA Leads

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

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

Trending Sources

Novo Nordisk Commits $285M to Ascendis to Kick Off Alliance on Longer-Acting GLP-1 Drugs

FDA Reconsideration Leads to Approval of Takeda Drug for Rare Esophagus Disorder

FDA setback leads women’s health biotech ObsEva to give up on uterine fibroids drug

New Hemophilia Data Lead FDA to Delay Decision for BioMarin Gene Therapy

After Data Miss in Lead Cancer Indication, Replimune Maps Path for Oncolytic Virus

Cardiac Complication Leads FDA to Place Foghorn Cancer Drug Under Partial Hold

Preclinical Safety Signal Prompts FDA Clinical Hold on Neumora Schizophrenia Drug

Vertex’s CRISPR Gene Therapy Lands Another FDA Nod in a Rare Blood Disease

Liver injury leads FDA to pause Sanofi’s tests of MS drug acquired in $3.7B deal

Amylyx ALS drug’s winding regulatory path leads to long-awaited FDA approval

In gene therapy race, bluebird leads Vertex in activating treatment centers but trails in patient starts

FDA Approves Bausch + Lomb Drug With a New Approach to Dry Eye Disease

FDA wants classwide boxed warning on all commercial CAR-T therapies amid secondary cancer safety probe

With FDA approval, Eli Lilly's weight-loss drug Zepbound arrives to challenge Novo Nordisk

Safety Risks and FDA Hold Lead Gilead to Stop Work on Drug in Blood Cancers

BMS, Mirati's Krazati cements lead over Amgen's Lumakras with FDA approval in colorectal cancer

FDA Approval of Travere Kidney Disease Drug Sets Up Blockbuster Expectations

FDA approves first-in-class oral antibiotic

RFK Jr. could 'cost lives in this country' if he pursues changes to US vaccine policy, Gottlieb says

Fierce Pharma Asia—BeiGene, Novartis' TIGIT divorce; Takeda's FDA withdrawal; Eisai's new Alzheimer's lead

Patient Death Leads to Pause in Test of 2seventy Bio Cell Therapy for Leukemia

By Eliminating Requirement for Animal Testing, FDA Modernization Act Allows Faster, More Cost-effective Drug Development

New FDA head needs to examine the accelerated approval program

AbbVie pays $255M to a buy biotech with drug candidate for fatal lung disorder

Amgen Osteoporosis Drug Prolia Is Hit With FDA’s Strictest Warning

Apellis Drug Becomes First Approved Therapy for Vision-Loss Disorder Geographic Atrophy

Eisai's US CEO and Alzheimer's lead Ivan Cheung telegraphs retirement days after Leqembi's full FDA nod

FDA’s ‘ivory tower thinking’ ignores promising biomarkers, says a rare disease CEO

Otsuka Commits $800M in M&A Deal Bringing a Phase 3-Ready Metabolic Disorder Drug

With FDA filing on the horizon, Karuna appoints new CEO

Regeneron hopes for quick FDA decision on high-dose Eylea as Roche's rival surges

An audiologist’s take on the news that OTC hearing aids are on their way to consumers

Here’s Why Novartis Is Paying $500M to Acquire a Preclinical RNA Startup

Applied Tx craters as FDA rejects lead product candidate

GSK's Arexvy maintains its RSV market dominance, looks to key label expansion as Moderna's mRNA threat looms

Pharma is big business whose goal is to maximize shareholder value

A scientific approach to irrational consumer choices

Bluebird touts Zynteglo, Skysona launch progress as FDA decision on sickle cell therapy nears

European authorities recognised for leading medicine regulatory oversight

The debate on DTC marketing is going to heat up again

Medical Devices Are Evolving Toward Specialized Solutions

FDA grants fast track status to FELIQS’s FLQ-101 to prevent ROP

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