Biopharma, 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

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

Biopharma

Biopharma FDA Leads

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

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

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

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

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

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

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

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

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

Biopharma sees strong Q2 2023 market cap growth

European Pharmaceutical Review

AUGUST 21, 2023

For 20 of the leading biopharmaceutical companies, the second quarter (Q2) of 2023 has brought an aggregate market capitalisation growth of 2.3 Q2 2023 biopharma market capitalisation results Eli Lilly “Eli Lilly witnessed the largest market capitalisation growth of 36.1 trillion in the Q1 of 2023 to $3.56 trillion in Q2 2023.

Biopharma

Biopharma Marketing Sales Medicine

$1.7b acquisition to develop rare haematology therapy

European Pharmaceutical Review

MAY 12, 2023

Swedish Orphan Biovitrum AB (Sobi ® ) has agreed to acquire CTI BioPharma for $1.7 The biopharma companies anticipate the transaction will help to change the treatment landscape for patients with rare diseases through new, innovative and effective medicines and therapies.

Food and Drug Administration

Food and Drug Administration Biopharma Medicine Food

Santhera seeks speedy FDA review of Duchenne drug vamorolone

pharmaphorum

OCTOBER 27, 2022

The Swiss biotech is seeking a priority review for vamorolone, which was licensed from US biotech ReveraGen BioPharma in 2020 after Santhera its former DMD therapy candidate idebenone failed clinical testing and was abandoned. Its lead drug has already claimed fast track and rare paediatric disease designations from the FDA.

FDA

FDA Side effects Biopharma Marketing

Pleco raises €17m for tumour microenvironment drug

pharmaphorum

SEPTEMBER 6, 2022

Pleco Therapeutics has closed a first-round financing, raising just over €17 million that it says will fund the development of its lead therapy for acute myeloid leukaemia (AML) through to regulatory filings in the US and EU. million in equity and R&D funding from Belgian biopharma company Hyloris, which was first announced last year.

Leads

Leads Biopharma Government Patients

Second phase 3 trial extends Astellas’ lead in claudin 18.2 cancers

pharmaphorum

DECEMBER 16, 2022

The FDA has already granted a fast-track designation to zolbetuximab for the treatment of gastric and GEJ adenocarcinoma. I-Mab Biopharma, meanwhile, has a bispecific antibody called TJ-CD4B – which binds to both claudin 18.2 The post Second phase 3 trial extends Astellas’ lead in claudin 18.2 Claudin 18.2

Leads

Leads Biopharma Prospecting Patients

Liver injuries prompt clinical hold on Sanofi BTK drug

pharmaphorum

JULY 1, 2022

billion buyout of Principia Biopharma has run into trouble, after the FDA placed it on partial clinical hold while a safety signal is investigated. Those changes haven’t been enough to prevent the FDA from taking action, although outside the US the studies will continue as planned with the tighter safety monitoring.

Side effects

Side effects Safety Biopharma Recruitment

AstraZeneca’s Tezspire for severe asthma gets green light in EU

pharmaphorum

SEPTEMBER 21, 2022

The European Commission has followed the lead of the US FDA and approved AstraZeneca’s Tezspire as an add-on maintenance therapy for patients with severe asthma, becoming the first and only biologic that can be used in all patients, and not restricted to those with specific forms of the disease.

Sales

Sales Patients Medicine Biopharma

Chiesi expands rare disease portfolio with Amryt Pharma acquisition

pharmaphorum

JANUARY 9, 2023

Biopharma and healthcare group Chiesi Farmaceutici S.p.A. Supported by voting agreements from leading Amryt shareholders and directors, and unanimously approved and recommended by the boards of both companies, the total transaction is worth up to $1.48 is to acquire the 2015-established Amryt Pharma Plc. on 6 th January.

Pharma

Pharma Biopharma Management Medicine

6 research and development life sciences predictions for 2023

pharmaphorum

JANUARY 6, 2023

With an early view of market viability, leading companies will be able to monetise new products quickly and at scale across Europe.”. This will enable biopharmas to create a more streamlined drug development process rooted in lean process execution and higher-quality data.”.

Safety

Safety Management Patients Training

How competitors are collaborating to shape biopharma best practices and regulations of the future

Pharmaceutical Technology

DECEMBER 20, 2022

The collaborative spirit arising between pharmaceutical and biotechnology companies is leading to a new rapport with regulators, as well. McCall sees the whole experience as a positive one for biopharma: “When I started in industry, this type of data sharing and collaboration was unheard of. Free Whitepaper.

Biopharma

Biopharma Manufacturing Pharmaceutical FDA

AstraZeneca on the hunt for new acquisitions

Pharmaceutical Technology

SEPTEMBER 5, 2022

On 23 August, at a Reuters Newsmakers online forum, Soriot announced his interest in increasing AstraZeneca’s portfolio of bolt-on acquisitions leading up to 2025. The FDA has granted the drug an orphan drug designation, and Phase III trials with eplontersen were completed In June. In 2019, AstraZeneca entered a $6.9

FDA

FDA Pharmaceutical Biopharma Pharma

Leading innovators in pyridine derivatives for the pharmaceutical industry

Pharmaceutical Technology

MAY 15, 2023

The US FDA database has 95 approved pyridine derivatives, including isoniazid and ethionamide (tuberculosis), delavirdine (HIV/AIDS), abiraterone acetate (prostate cancer), and tacrine (Alzheimer’s).

Pharmaceutical

Pharmaceutical Leads Pharmacology Medicine

PM360 2024 Innovative STARTUPS: Courier Health

PM360

DECEMBER 20, 2024

Courier Health Powered by machine learning AI, Avalere Courier Health was founded in 2021 with the goal of revolutionizing the biopharma landscape with its groundbreaking patient-focused customer relationship management (CRM) platform. It simplifies complex workflows (e.g., In July 2024, Courier Health secured $16.5

Customer Relationship Management

Customer Relationship Management CRM Biopharma Relationship management

Meeting rising demands of a new radiotheranostic era

European Pharmaceutical Review

AUGUST 31, 2023

Compared to the aforementioned copper‑based radionuclides, Cu-64 has a longer production and decay time, which leads to a more expensive production process and a higher radiation burden for the patient. During her career, she held positions of increasing responsibility to lead companies in the field, including AREVA/Framatome.

Food and Drug Administration

Food and Drug Administration Manufacturing Safety Transportation

The use of genomic profiling testing to improve oncology care

pharmaphorum

SEPTEMBER 12, 2022

Dr Prasanth Reddy, head of oncology at LabCorp, tells us how the company works with cancer care teams, biopharma partners, and patients as an integral part of the patient journey from testing to treatment. They offer the only diagnostic kit cleared by the US FDA for pan-solid cancer comprehensive tumour profiling using a 500+ gene panel.

Patients

Patients Medicine FDA Healthcare

From viral vectors to nucleic acids: biopharma’s evolving manufacturing landscape

European Pharmaceutical Review

APRIL 30, 2024

What are the top three trends that you are seeing in the biopharma manufacturing space? Let’s talk about continuous biopharma manufacturing. Speaking of your biopharma clients, what are you hearing about their main priorities? I believe this is going to be an emerging trend in the market.

Manufacturing

Manufacturing Food and Drug Administration Biopharma Marketing

Shorter timelines, evolving strategies: Four key trends in regulatory approvals of new medicines

Clarivate

AUGUST 2, 2022

Four major trends came to light from CIRS’ most recent analysis of new active substance (NAS) approvals for the European Medicines Agency (EMA), US Food and Drug Administration (FDA), Japanese Pharmaceuticals and Medical Devices Agency (PMDA), Health Canada, Swissmedic and the Australian Therapeutic Goods Administration (TGA).

Medicine

Medicine Food and Drug Administration FDA Healthcare

Targeting tumours with novel radiopharmaceuticals

European Pharmaceutical Review

OCTOBER 19, 2023

billion deal, in which Eli Lilly agreed to acquire two of POINT Biopharma Global’s late-stage radioligand therapies for oncological indications. Can you share an update on the clinical trial for your lead candidate? Hoppin served as lead director of Noria as the two organisations were sold to Bayer Healthcare in June of 2021.

Food and Drug Administration

Food and Drug Administration Manufacturing Side effects Doctors

Low-Priced Oncology Disruptors: The Cuckoo in the PD-1/PD-L1 Nest or the Runt of the Clutch?

pharmaphorum

SEPTEMBER 30, 2022

Perhaps more interestingly, similar signals have also emerged from traditional biopharma heavyweights, such as Novartis and Lilly, which one would typically associate with more conservative approaches to pricing. New manufacturers, such as EQRx, have been vocal in their desire to bring new PD-1/PD-L1 checkpoint inhibitors (e.g.

Consulting

Consulting Marketing Pharma Manufacturing

Untangling the Complexities of Cell and Gene Therapy Clinical Trials: A Supply Chain Perspective

Pharmaceutical Technology

MAY 24, 2023

Every major pharma company is now involved in CGT development which has resulted in the approval of 28 therapies by the FDA thereby making CGT no longer a niche category of therapies. This major difference leads to very different requirements and processes for the clinical trial supply chain, which are summarized below.

Manufacturing

Manufacturing Transportation Specialization Patients

Alpha TAU Killing Tumors With Highly Targeted Alpha Radiation

Medgadget

MARCH 2, 2023

This leads to substantial damage to healthy tissues and too often results in poor outcomes. The FDA looked at the data, saw it promising, and asked for a small feasibility study in the U.S. We hope to see Alpha TAU presenting its technology at Biomed Israel , the leading international Life Science and HealthTech conference in Israel.

Manufacturing

Manufacturing Leads Patients Medicine

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

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

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

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

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

FDA Approval of Travere Kidney Disease Drug Sets Up Blockbuster Expectations

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

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

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

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

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

Biopharma sees strong Q2 2023 market cap growth

$1.7b acquisition to develop rare haematology therapy

Santhera seeks speedy FDA review of Duchenne drug vamorolone

Pleco raises €17m for tumour microenvironment drug

Second phase 3 trial extends Astellas’ lead in claudin 18.2 cancers

Liver injuries prompt clinical hold on Sanofi BTK drug

AstraZeneca’s Tezspire for severe asthma gets green light in EU

Chiesi expands rare disease portfolio with Amryt Pharma acquisition

6 research and development life sciences predictions for 2023

How competitors are collaborating to shape biopharma best practices and regulations of the future

AstraZeneca on the hunt for new acquisitions

Leading innovators in pyridine derivatives for the pharmaceutical industry

PM360 2024 Innovative STARTUPS: Courier Health

Meeting rising demands of a new radiotheranostic era

The use of genomic profiling testing to improve oncology care

From viral vectors to nucleic acids: biopharma’s evolving manufacturing landscape

Shorter timelines, evolving strategies: Four key trends in regulatory approvals of new medicines

Targeting tumours with novel radiopharmaceuticals

Low-Priced Oncology Disruptors: The Cuckoo in the PD-1/PD-L1 Nest or the Runt of the Clutch?

Untangling the Complexities of Cell and Gene Therapy Clinical Trials: A Supply Chain Perspective

Alpha TAU Killing Tumors With Highly Targeted Alpha Radiation

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