The Commissioner’s National Priority Vouchers aim to offer accelerated pathways to drugs that meet certain criteria, perhaps including a low price-tag. But the policy is vaguely defined and was announced without public input, going against the FDA’s own published practices, experts say.
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.
The European Union’s health regulatory agency did not endorse approving Elevidys for ambulatory patients with Duchenne muscular dystrophy.
The strategic reprioritization comes after the company hit two major hurdles in the past year, including a clinical hold for an investigational gene therapy and an FDA rejection for its lead asset.
CBER is unanimously against Elevdiys’ return to the market without additional evidence, according to media reports citing an anonymous senior FDA official. Given Elevidys’ full approval, however, experts told BioSpace this path would set up a length legal battle between the regulator and Sarepta Therapeutics.
The company didn’t share specific data for the molecule, gefurulimab, but said it hit all endpoints in the Phase III PREVAIL trial and promised to share more at an upcoming scientific meeting.
FEATURED STORIES
Approaches and targets for depression and other mental health illnesses have remained stagnant for decades. With several readouts for novel therapies on the horizon, that could be changing.
Despite weathering a difficult year, biopharma continues to see massive pay gaps between CEOs and their median employees, with top executives often earning hundreds of times more.
Until compelling surface targets for lung cancer are developed, antibody-drug conjugates will fail to treat most patients with lung cancer, experts told BioSpace.
The insights AI affords can potentially boost sustainability, but it’s unclear whether these gains outweigh the technology’s environmental cost.
Following a series of clinical failures, optimism builds for the first disease-modifying treatment.
Since taking the helm in 2023, Anderson has embarked on a radical departure from the traditional structure of large pharma companies. Will getting rid of management layers fix Bayer?
LATEST PODCASTS
In this episode presented by Slone Partners, Leslie Loveless, Co-CEO and Managing Partner discusses how hiring and the building of executive teams has responded to the current biotech environment.
Pfizer seals the deal with Metsera for $10 billion after Novo Nordisk bowed out; President Donald Trump welcomes executives from Novo and Eli Lilly to the White House to announce that the companies’ GLP-1 medicines would be sold at a reduced cost; and the FDA grants the second round of priority review vouchers—primarily to already marketed drugs.
In this episode presented by PII, BioSpace’s head of insights discusses how to relieve clinical trial patients of technological burden to improve compliance with guests Oliver Eden and Travis Webb.
Job Trends
Kite Receives U.S. FDA Approval of Manufacturing Process Change Resulting in Reduced Median Turnaround Time for Yescarta ® CAR T-cell Therapy.
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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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Novel therapies often pass through several owners on their way to the market. Here’s a look at some of the drugs that got dropped before they hit primetime. -
Japanese biopharma scoops up Iveric’s investigational drug for age-related blindness disease. The drug, which trails Apellis’ Syfovre, is awaiting FDA approval with a decision expected by August. -
The acquisition of Bellus Health will give GSK access to camlipixant, a potentially best-in-class P2X3 antagonist for chronic cough. -
Merck finalized the acquisition of immune-focused Prometheus Biosciences for approximately $10.8 billion, picking up mid-stage ulcerative colitis and Crohn’s asset. -
BioNTech will pay Shanghai-based Duality Biologics $170 million upfront for rights to two of its topoisomerase-1 inhibitor-based antibody drug conjugates.
WEIGHT LOSS
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Over the past year, obesity treatments have become the focus of intense media attention and discussion in healthcare — with increased focus on Wegovy and Zepbound. This attention has highlighted the need to address the global obesity epidemic. -
Eli Lilly has signed a multi-year contract with animal genomics biotech Fauna Bio to use its artificial intelligence platform to discover drug targets for obesity in a deal worth nearly half a billion dollars. -
A GLP-1-based drug from Structure Therapeutics shows clinical promise but not enough for some on Wall Street. -
The study found only 19% of nearly 2,000 patients were still taking their prescribed anti-obesity medication at 12 months, compared to 40% that continued with Novo Nordisk’s Wegovy. -
This week we discuss ups and downs in the weight loss and ADC markets - including Altimmune, Pfizer setbacks, AbbVie’s ImmunoGen buy and more.
POLICY
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After back-to-back failures in 2021, Wave Life Sciences has finally aced a Phase Ib/IIa Huntington’s disease trial and is looking to a potential accelerated approval for its investigational antisense oligonucleotide. -
Scrutiny of WuXi Raises Potential Opportunities for Indian CDMOsAs congressional pressure increases on WuXi AppTec and other China-based companies over alleged ties to the Chinese government, India’s contract development and manufacturing organization sector could benefit. -
Citing issues with a third-party manufacturer, the FDA has issued another Complete Response Letter to AbbVie rejecting its New Drug Application for ABBV-951, a proposed treatment for motor fluctuations in adults with advanced Parkinson’s disease. -
If approved, ensifentrine would be the first non-steroidal, anti-inflammatory drug for patients with chronic obstructive pulmonary disease, offering an option with potentially fewer side effects. -
The FDA’s calendar is relatively light in July, with only five major deadlines, including one for a PD-1 blocker and another for an opioid overdose drug.
In this graceless age where people look at their phones when you are talking to them, only communicate via text or email, scroll head down on their phone while a speaker is presenting, post inappropriate pictures and commentary on Facebook, it’s no wonder many people do not send thank you notes.
Have you ever experienced a career epiphany but lacked the resolve to act on it? Don’t feel bad, especially if “acting on it” meant leaving a secure, albeit unfulfilling, job for a new career in a different industry.
When you are constantly hearing, “Thank you for your time, but we went with another candidate,” or you’re regularly submitting applications online, but not ever getting a response back, it can be frustrating.
The moment you step on the job market, you’re selling yourself. Yes, you, the job seeker, are a “brand,” and each touchpoint you have with a potential new employer is an opportunity to “sell” that brand, that vision of your candidacy, the promise of yourself as a future team member and employer.
Not only are interviewers tasked with evaluating whether or not you’re a good fit for their organization and the role, but you also should take every opportunity you can to evaluate if the position, company, and team is a good match for your own career goals and needs.
Career paths are not always perfectly linear and uninterrupted. Here are some ways you can display gaps in resumes that don’t appear like a weak point.
HOTBEDS
REPORTS
In this Employment Outlook report, BioSpace explores current workforce sentiment, job activity trends and the prospective job and hiring outlook for 2025, particularly as it compares to the previous year.
BioSpace’s third report on diversity, equity, inclusion and belonging in life sciences examines dramatic shifts in attitude around diversity initiatives.
CANCER
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Genmab announced Wednesday it is buying ProfoundBio and its pipeline of next-generation antibody-drug conjugates being developed for gynecologic cancers and other solid tumors. -
With second-generation antibody-drug conjugates, Eli Lilly, Daiichi Sankyo and others look to reduce toxicity and improve the magnitude and duration of response. -
Avenzo’s founders, who led Turning Point through its $4.1 billion sale to BMS, want to in-license three clinical-stage oncology candidates by early 2026. -
Bristol Myers Squibb’s $4.8 billion acquisition of Mirati pays off with strong data from the KRYSTAL-12 study of Krazati, showing that the KRAS inhibitor significantly improves progression-free survival. -
Keytruda can now be used in the European Union for patients with resectable non-small cell lung cancer at high risk of recurrence in combination with platinum chemotherapy, then continued as a monotherapy afterwards.
NEUROSCIENCE
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BIIB080 successfully reduced tau pathology in patients with early-stage disease across all six brain regions analyzed. -
Data released Wednesday from the Phase III A4 study showed solanezumab fell short of its primary endpoint, failing to slow cognitive decline in patients with preclinical Alzheimer’s disease. -
The FDA set a decision date of July 6, 2023 for Eisai and Biogen’s recently approved Alzheimer’s drug, Leqembi, Eisai announced Monday. -
Billy Dunn, director of the FDA’s Office of Neuroscience, is stepping down from his post to “explore other opportunities,” according to an internal FDA e-mail. -
If Leqembi is granted priority review from the agency, full approval could come as soon as July, Ivan Cheung, chairman and CEO, Eisai Inc., told CNBC Thursday.
CELL AND GENE THERAPY
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Through substantial leadership turnover and workforce cuts, the FDA has continued to support the advanced therapy sector, actively working to remove obstacles to innovation. -
The plausible mechanism pathway “could accelerate gene therapy/editing development,” analysts at William Blair said Thursday, while adding that additional clarity is needed. -
MeiraGTx Holdings is licensing a genetic eye disease medicine to Eli Lilly in a deal worth up to $475 million. -
The FDA previously placed two clinical studies on hold, including the Phase III trial in which the liver toxicity occurred. Intellia is working with experts to create a risk management program for nex-z. -
Pfizer and Novo Nordisk continue to fight for ownership of obesity startup Metsera; CDER Director George Tidmarsh leaves his position amid an ongoing probe into his “personal conduct”; FDA reverses course on approval requirements for uniQure’s Huntington’s gene therapy; Sarepta’s exon-skipping Duchenne muscular dystrophy drugs fail confirmatory study.
