A week into his tenure as head of the FDA’s Center for Drug Evaluation and Research, experts agree that Rick Pazdur is the “ideal fit” to stabilize the agency. And, according to one ex-FDA official, if his CBER counterpart Vinay Prasad tries to supersede Pazdur’s authority, “there will be hell to pay.”
“We felt we had a responsibility to explore semaglutide’s potential, despite a low likelihood of success,” Martin Holst Lange, Novo’s R&D chief, said on Monday.
NervGen will meet with the FDA early next year to align on a regulatory path forward for NVG-291 in chronic spinal cord injury.
Asundexian’s Phase III win could also bode well for Bristol Myers Squibb, which is also developing a Factor XIa inhibitor called milvexian for stroke prevention, analysts said.
A source familiar with the matter said the White House initially requested the resignation of Sanjula Jain-Nagpal, a policy and research official at the FDA.
The regulator has received reports that a group of patients treated with Adzynma had neutralizing antibodies against the protein the therapy replaces.
After revoking Sarepta’s award in July and awarding one to Krystal last month, the FDA’s platform technology designation program appears to be back on track. These six biotechs could be on the regulator’s radar.
FEATURED STORIES
Drug pricing criticism often fixates on a price at a single point in time but drug pricing is never static.
Cell and gene therapy experts question where the FDA designation fits in an environment that features a range of intersecting regulatory perks.
As industry leaders gather at the annual event in Phoenix, the cell and gene therapy space remains in a state of flux, with M&A activity and regulatory support signaling momentum while commercialization challenges continue to hinder broader investor interest.
Smarter design through targeted delivery and human-relevant testing can save the industry from costly safety failures.
While Bruton’s tyrosine kinase inhibitors are often hailed as the next big breakthrough in multiple sclerosis, Immunic Therapeutics and others are leveraging neuroprotective targets and remyelination to keep the disease at bay.
Following up on previous, dimly received issuances, a new set of ideas published by the FDA to streamline regulatory pathways for cell and gene therapies ‘for small populations’ is receiving a warmer welcome—but experts warn it will take more to turn the tide for the fraught therapeutic space.
LATEST PODCASTS
Sarepta Therapeutics faces serious FDA action after news broke of a third patient death, the FDA gets a new top drug regulator in George Tidmarsh, a handful of new drugs get turned away from the market and pharma companies continue to commit billions to reshoring manufacturing.
In this episode presented by Eclipsebio, BioSpace’s head of insights Lori Ellis continues the discussion on mRNA and srRNA with Andy Geall of Replicate Bioscience and Alliance for mRNA Medicines and Pad Chivukula of Arcturus Therapeutics.
In this episode presented by IQVIA, BioSpace’s head of insights Lori Ellis discusses the evolving role of local qualified persons for pharmacovigilance with Ana Pedro Jesuíno, global head local QPPV network at IQVIA.
Job Trends
The headcount reduction will save money that the company will use in developing mavorixafor, its CXCR4 antagonist that last year received FDA approval to treat WHIM syndrome, in the larger patient population with chronic neutropenia.
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SPECIAL EDITIONS
In this deep dive, BioSpace investigates China’s rise as a biotech powerhouse.
In this deep dive, BioSpace explores the next big thing in obesity.
BioSpace did a deep dive into biopharma female executives who navigated difficult markets to lead their companies to high-value exits.
DEALS
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Sarepta’s troubles had nothing to do with Arrowhead’s assets, and yet both companies have seen their stock prices decline this past month. BioSpace caught up with Arrowhead’s Chris Anzalone to talk about the biotech’s role as an RNAi pipeline savior. -
Out-licensing drugs to multinational corporations is a natural step for Chinese biotechs, but the recent rise in deals is only scratching at the surface of partnership-ready biotechs in the region. -
The German giant is looking to develop new drugs for undisclosed eye diseases using Re-Vana’s extended-release injectable platform to supply drugs to the eye for months at a time. -
The star of GSK’s Hengrui partnership is the COPD candidate HRS-9821, which will complement the pharma’s respiratory pipeline that’s anchored by the anti-asthma drug Nucala. -
The collaboration focuses on ‘molecular gates,’ a class of molecules that the startup company Gate Bioscience says can stop pathogenic proteins from leaving the cell.
WEIGHT LOSS
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As Novo Nordisk cuts 9,000 people from its organization in a restructuring effort, BioSpace looks back on the Danish pharma company’s rise. -
This week’s release of the Make America Health Again report revealed continued emphasis on vaccine safety; Health Secretary Robert F. Kennedy Jr.’s faceoff with senators last week amounted to political theater; the FDA promises complete response letters in real time and shares details on a new rare disease framework; and Summit disappoints at the World Conference on Lung Cancer in Barcelona. -
Suddenly one obesity asset has come to define Amgen but executives see a fuller portfolio that will bring the big biotech into the future. -
While a win for consumers, the regulatory action did nothing to stem the manufacture of compounded versions of the popular obesity drugs that are made by Novo Nordisk and Eli Lilly. In fact, the FDA seems to be signaling that “some level of compounded product is acceptable,” according to BMO Capital Markets. -
The World Health Organization’s Essential Medicines list guides high-level procurement and coverage decisions for over 150 countries.
POLICY
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In coordination with the United States President’s Emergency Plan for AIDS Relief, Gilead will make its twice-yearly HIV prophylactic Yeztugo available to resource-limited countries “at no profit.” -
Perhaps the most interesting of the pile of FDA rejection letters was for Lykos Therapeutics’ MDMA therapy. Letters sent to Stealth BioTherapeutics, Regeneron and more were also released as the agency also promised future CRLs “promptly after they are issued to sponsors.” -
The new additions would bring ACIP membership to 14 total. Several of the proposed members have taken part in anti-vaccine activity or made anti-vaccine statements. -
YouTube has shut down a channel containing hundreds of videos of comments made by doctors and other influencers—including CBER Director Vinay Prasad, Health Secretary Robert F. Kennedy Jr. and NIH Director Jay Bhattacharya—during the pandemic. This comes as Prasad reveals further details about last week’s updated COVID-19 approvals. -
Albert Bourla heralded the president’s COVID-19 leadership and Operation Warp Speed initiative as a Nobel Prize–worthy achievement and said that Pfizer stands by the integrity of the data already shared.
Companies will look at job candidates’ LinkedIn profiles, so make sure yours is strong, from the summary of your expertise and qualifications to testimonials from colleagues.
After more than 20 years at Eli Lilly, Leslie Sam moved into independent consulting. To prepare for the transition, she focused on becoming technically deep and earning industry recognition.
Employed and unemployed biotech and pharma professionals are thinking about job hunting in other fields amidst a challenging labor market.
When you don’t get the promotion you wanted, it’s important to assess your company and yourself so you can improve your odds in the future.
Looking for an automation engineer job? Check out these seven companies hiring life sciences professionals like you.
Career support is the No. 2 driver of employee engagement while learning and development is No. 3, according to a new Right Management report. A recent BioSpace survey supports those findings.
HOTBEDS
REPORTS
The 2021 Salary Report is significant in more ways than one. This year, BioSpace analyzed salary data through two key lenses: the impact of the pandemic, and for the first time, race and ethnicity.
BioSpace’s 2022 Salary Report explores the average salaries and salary trends of life sciences professionals. Though movement in the labor market slowed during the pandemic, recovery has been swift and employers are once again having to cope with a highly competitive talent market.
BioSpace has created guidelines on how biopharma organizations, large and small, can effectively support employee resource groups (ERGs), encouraging both their creation and ongoing participation.
CANCER
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After two patients who received the investigational CDC7 blocker died, pushing forward with SGR-2921’s development would be “difficult,” according to Schrödinger, whose stock dropped 17.5% before the opening bell on Thursday. -
For $1.3 billion in aggregate—including upfront and milestone payments—Bayer will get exclusive global access to Kumquat Biosciences’ small-molecule KRAS G12D blocker. -
Phase Ib data show Hernexeos can elicit a confirmed objective response rate of 44% in patients with HER2-mutated NSCLC who had previously been treated with a directed antibody-drug conjugate. -
The recent announcement of RFK Jr.’s termination of mRNA vaccine contracts is the latest effort to undermine this promising technology at the federal level. Pharmaceutical companies and private investors must fill the gap and ensure that research into this critical resource continues. -
Arguably the FDA’s most anticipated decision this month is for a subcutaneous induction formulation of Biogen and Eisai’s Alzheimer’s drug Leqembi, which, according to Eisai, could “help reduce the burden on healthcare professionals and patients.”
NEUROSCIENCE
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After a demoralizing period punctuated by the withdrawal of one of the few marketed therapies for ALS, investment in new biotechs, state-backed collaborative initiatives and buzz at BIO2025 suggest a new day in drug development for one of medicine’s most intractable diseases. -
In another blow to Prothena’s neurodegenerative disease portfolio, anti-amyloid candidate PRX012 has run into the same problem that larger peers Biogen and Eli Lilly have battled: high rates of swelling in the brain. -
AMX0035—approved as Relyvrio in 2022 for amyotrophic lateral sclerosis but voluntarily pulled from the market last year—was unable to distinguish itself from placebo in a mid-to-late-stage trial of progressive supranuclear palsy. -
Novartis has bet up to $772 million to gain access to BioArctic’s BrainTransporter platform, which was leveraged in a partnership with Eisai to produce Leqembi. -
Eli Lilly drops a second Phase III readout for orforglipron; AbbVie committed to the psychedelic therapeutics space with the $1.2 billion acquisition of Gilgamesh’s depression asset; the CDC taps vaccine skeptic Retsef Levi to lead its COVID-19 immunization working group; and the FDA prioritizes overall survival in cancer drug development.
CELL AND GENE THERAPY
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In the wake of multiple patient deaths from liver injuries related to Sarepta Therapeutics’ AAV gene therapy platform, some in the sector are looking for ways to improve the current technology, while others are eager to move on. -
Sarepta did not hold an investor call for its second-quarter earnings report or provide an updated full-year revenue outlook. -
From innovation in manufacturing to more-flexible regulation and better communication with payers, much needs to happen to make CGTs commercially viable. But it is possible, experts agreed at a recent panel. -
The primary focus in scaling up production should first be the adoption of lean manufacturing principles used in virtually every other industry. -
The pivotal Phase II trial is testing Allogene’s CAR T candidate cemacabtagene ansegedleucel for large B-cell lymphoma. ALLO-647 was being used as a preparative lymphodepletion therapy.
