The star of the acquisition is the enzyme replacement therapy INZ-701, being developed for the rare disease ENPP1 deficiency.
The FDA also approved the use of Zynyz as a monotherapy for patients with squamous cell carcinoma of the anal canal who are intolerant to platinum chemotherapy or whose disease has progressed.
Since Elevidys’ accelerated approval in 2023, experts have been clamoring for more data, particularly in older and non-ambulatory children. New results, presented Friday, show mobility improvements in 8- to 9-year-old patients after one year of follow-up.
Lilly will use Rznomics’ proprietary ribozyme technology to develop RNA editing therapies for congenital hearing loss.
Over Lars Fruergaard Jørgensen’s eight years as CEO, Novo’s sales, profits and share price have almost tripled, the company said. However, the shares have taken a turn since mid-2024, falling by half in one year.
As the FDA prepares for a busy Oncologic Drugs Advisory Committee meeting next week, an agency insider told BioSpace that volunteers with little training are scrambling to secure the required expertise after workforce cuts decimated the adcomm planning office.
FEATURED STORIES
While Thursday’s label expansion and traditional approval for the gene therapy is an important milestone, many challenges still face the Duchenne muscular dystrophy community.
On the heels of a Phase III flop for Pfizer’s Duchenne muscular dystrophy gene therapy candidate, the FDA has green lighted the expanded use of Sarepta Therapeutics’ Elevidys.
The pharma industry is staring down the barrel of a widespread loss of exclusivity, with more than 190 products going off-patent between 2022 and 2030. Here are some strategies company are employing to manage the drop in revenue.
Four executives with collective decades spent at BMS, Roche, Astellas, Eli Lilly and more gave their insights on navigating a biopharma career during a Monday DIA panel in San Diego.
The next six months for the FDA are primed to be as groundbreaking as the first six, with Eli Lilly’s donanemab and Lykos Therapeutics’ MDMA-assisted PTSD therapy on the docket, among others.
The FDA is facing four big target action dates in the final week of June, including one label expansion for a bispecific antibody and another for an investigational gene therapy.
FROM BIOSPACE INSIGHTS
While FDA Commissioner Marty Makary emphasizes learning and humility, the FDA has systematically removed the very experience that would make change possible.
LATEST PODCASTS
In this episode presented by IQVIA, BioSpace’s head of insights Lori Ellis discusses the concerns and opportunities of patient data driving AI tasks with Louise Molloy, associate director medical information and pharmacovigilance.
Sarepta’s Elevidys is back on the market for ambulatory patients with Duchenne muscular dystrophy, Health Secretary Robert F. Kennedy Jr. reportedly plans to dissolve the U.S. Preventive Services Task Force and “fix” the vaccine injury compensation program, Merck, AstraZeneca and more report Q2 earnings, Novo names a new leader and Roche’s trontinemab impresses at AAIC25.
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.
Job Trends
CSL Vifor announced that Health Canada has authorized Ferinject for the intravenous treatment of iron deficiency anemia in adult and pediatric patients one year of age and older when oral iron preparations are not tolerated or are ineffective, as well as for the treatment of iron deficiency in adult patients with heart failure and New York Heart Association class II/III* to improve exercise capacity.
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SPECIAL EDITIONS
BioSpace did a deep dive into executive pay, examining the highest compensation packages, pay ratios and golden parachutes—what a CEO would get paid to leave.
A new generation of checkpoint inhibitors is emerging, with some showing more promise than others. From recent TIGIT failures to high-potential targets like VEGF, BioSpace explores what’s on the horizon in immuno-oncology.
Peter Marks, the venerable head of the FDA’s Center for Biologics Evaluation and Research, has been forced out. In this special edition of BioPharm Executive, BioSpace takes a deep dive into the instability of the HHS.
DEALS
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The Japanese biotechnology and food company has bought into the gene therapy space with its $620 million acquisition of Ohio-based CDMO and clinical-stage biotech Forge Biologics. -
The cell therapy-focused biotech will use most of the net proceeds from its initial public offering to fund Phase II clinical trials for its lead program, a novel CAR T-cell candidate. -
The New York-based genetic medicine company, which expects gross proceeds of approximately $100 million, joins a small group of biotechs that have launched initial public offerings this year. -
The Swiss drugmaker gains rights to RVT-3101 in the U.S. and Japan. Telavant was formed in late 2022 by Roivant and Pfizer, which had a 25% stake in the venture and retains rights to the antibody in other countries. -
While Merck lost out to Pfizer earlier this year in snapping up Seagen, this week the company closed a deal worth a potential $22 billion with Daiichi Sankyo—further evidence of the industry’s insatiable appetite for ADC technology.
WEIGHT LOSS
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Despite strong demand for weight-loss drugs, the lack of Medicare coverage is potentially interfering with prescription and dispensing rates of anti-obesity medications for elderly adults, according to a new study. -
Sanders says he wants Novo Nordisk to “do the right thing” and lower the costs of Ozempic and Wegovy. But only the Inflation Reduction Act can achieve that. -
Now in Phase III with its small molecule orforglipron, Eli Lilly is leading the oral GLP-1 race against Novo Nordisk, Pfizer, Roche and others. -
Altimmune on Wednesday said it is ending development of HepTcell, a hepatitis B candidate, following disappointing trial results as it focuses on obesity and metabolic dysfunction-associated steatohepatitis. -
Citing a JAMA study that found Ozempic could be profitably produced at under $5 per month, Senator Bernie Sanders on Wednesday called on Novo Nordisk to lower prices for the diabetes treatment and the weight-loss drug Wegovy.
POLICY
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After discontinuing its long-acting insulin product Levemir, Novo has again found itself under legislative scrutiny, with three Democratic senators seeking a sit-down with the pharma. -
Vertex has filed a complaint against the Department of Health and Human Services, seeking to make its fertility preservation program available to federally insured patients needing Casgevy treatment. -
Why I advocated on Capitol Hill this month for the renewal of the FDA’s Priority Review Voucher program -
The Federal Trade Commission criticized the business practices of pharmacy benefit managers this week, but drugmakers are also at fault for the high costs of medicines.
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The Senate on Thursday unanimously passed a bill aimed at limiting the number of patents drugmakers can introduce and making it easier for generic and biosimilar competitors to enter the market.
Two effective focal components – deployed separately or together – are the resume headline and resume branding statement.
Going into an interview knowing that it’s OK to be yourself and perfection isn’t the goal can help alleviate that self-imposed pressure.
In order to play the “cat and mouse” game with poise, we’ve provided a breakdown of the steps to take to negotiate properly. Read on for tips to help you successfully negotiate a job offer.
Once you’ve determined where you want to reside, the next step is hunting for a premier job in your field in that location.
Before you even begin researching, prioritize which aspects of the job offer are most important to you.
First, prioritize these criteria according to your own desires and needs. Which aspects of the offer do you most value?
HOTBEDS
IN CASE YOU MISSED IT
BPL-003 showed “robust” efficacy data in treatment-resistant depression, according to analysts from Jefferies, who noted that the asset could hit peak market sales of $1 billion. The results clear the way for the asset’s late-stage development and for the completion of a proposed merger with atai Life Sciences.
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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Novartis continues its dealmaking spree with a bet on Dren Bio’s Targeted Myeloid Engager and Phagocytosis platform to develop bispecific antibodies in oncology. -
A longtime biopharma exec and Moderna shareholder argues in an anonymous email to the companies’ CFOs that they have a fiduciary responsibility to close the deal. Analysts say the proposal is interesting but “too simplistic.” -
AstraZeneca is proposing to use Imfinzi for resectable non-small cell lung cancer in the perioperative setting—both before and after surgical resection of the tumor. However, ahead of Thursday’s advisory committee meeting, FDA staffers have expressed concerns. -
Third Arc Bio, led by three former Johnson & Johnson executives, is advancing a portfolio of multifunctional antibodies for cancer and inflammatory and immune-mediated diseases. -
GSK is seeking to relaunch Blenrep after its market withdrawal in 2022. The pharma is eyeing a second-line niche for the antibody-drug conjugate in the treatment of relapsed or refractory multiple myeloma.
NEUROSCIENCE
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Contineum Therapeutics joined the 2024 initial public offering class on Friday with an SEC filing. The biotech will use the IPO proceeds to complete a Phase II trial for its most mature candidate targeting multiple sclerosis. -
Heather, Greg and Tyler discuss a busy news week including Wegovy’s label expansion, biosimilars, surprise donanemab delays for Eli Lilly and speculate on election impact. -
The FDA plans to convene an advisory committee meeting to discuss the safety profile and efficacy of Eli Lilly’s Alzheimer’s candidate, the company announced Friday. -
The regulator’s new draft guidance, released on Monday, provides additional details regarding the use of surrogate and biomarker endpoints to support accelerated approvals. -
In Monday’s neurology update for investors, Roche touted data from a small Ib/IIa trial for trontinemab, an investigational therapy for Alzheimer’s disease which demonstrated “rapid and robust” amyloid plaque reduction.
CELL AND GENE THERAPY
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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. -
The FDA greenlit multiple new drugs this month and issued some notable label expansions, including for Eli Lilly’s Kisunla. Meanwhile, the regulator turned away a cell therapy for Duchenne muscular dystrophy and a gene therapy for the rare disease Sanfilippo syndrome.
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As analysts parsed news of Vinay Prasad’s ouster, worries over drug approval delays, cell and gene therapy impacts and more were top of mind. -
The swift FDA action removes an overhang from Sarepta and allows Elevidys to return to the market without another safety study, as had been feared, Jefferies analysts said Monday. -
Brazilian authorities said the death was unlikely to have been caused by Elevidys and was instead more in line with severe infection exacerbated by immunosuppression.
