FDA Leucovorin Approval Raises Concerns About Reliance On Case Reports
The FDA granted approval for leucovorin calcium tablets to treat a rare genetic disorder that impairs folate transport to the brain. Unlike typical approvals, the agency relied primarily on a handful of patient case reports rather than a sponsor‑run clinical trial. The decision has sparked criticism from health‑policy analysts who question the evidentiary foundation. The approval expands treatment options for a small patient population while raising regulatory eyebrows.
STAT+: GOP Senator Is Investigating the FDA over Rejections of Rare Disease Drugs
Sen. Ron Johnson (R‑Wis.) announced an investigation into the FDA’s rejections of rare‑disease treatments, requesting the agency’s complete response letters for drugs targeting ataxia, Sanfilippo syndrome and similar conditions. Johnson argues the FDA’s cited deficiencies are often “nitpicky,” suggesting an...
Biologics and Biosimilars Landscape 2025: IP, Policy, and Market Developments
The FDA approved 18 biosimilars in 2025, spanning six therapeutic areas and marking a surge in interchangeable designations to over 20. A wave of denosumab biosimilars and first‑in‑kind interchangeable products such as Poherdy® and Omlyclo® highlighted market diversification. BPCIA litigation...
Public Citizen To Push Compulsory Licensing If Pfizer Withholds Drugs From France
Public Citizen announced it will encourage compulsory licensing of generic drugs if Pfizer follows through on its January threat to withhold medicines from France unless the country raises drug prices. The consumer group’s stance turns a pricing dispute into a...
Study Suggests Long Non-Coding RNA Has Potential as New Class of Genetic Medicine
Researchers at the University of Toronto have engineered synthetic long non‑coding RNA (lncRNA) molecules that can dampen inflammatory responses. The study, led by PhD student Janice Pang and biologist Omar Khan, demonstrates that these engineered lncRNAs reduce cytokine release in...
CDMO Neuland Labs Expects Commercial Production Facility to Be Operational by Summer
Neuland Laboratories announced that the first module of its new commercial peptide manufacturing facility will be operational by summer on its 17‑acre Bonthapally campus in India. The module adds 6,370 L of solid‑phase and liquid‑phase peptide synthesis capacity, ranging from 250 L...
Lundbeck CEO Talks Drug Pricing, Protecting Biotech and European Needs
Lundbeck chief executive Charl van Zyl warned that Europe’s fragmented drug‑pricing regime threatens biotech sustainability and patient access. He cited recent remarks by HHS Secretary Robert F. Kennedy Jr. suggesting Europeans already pay higher prices than Americans. Van Zyl called...
FDA Clears New Formulation of Lantheus' PSMA Imaging Agent
The FDA has cleared Pylarify TruVu, a reformulated version of Lantheus’ PSMA‑targeting radiopharmaceutical piflufolastat F‑18. The new formulation improves stability at higher radioactive concentrations, enabling larger batch production and broader distribution. Lantheus plans a Q4 2026 commercial launch with a rolling geographic...
Phase III Data Support Johnson & Johson’s Teclistamab as Second-Line Therapy for RRMM
Johnson & Johnson has filed a Type II variation with the European Medicines Agency to add teclistamab, a bispecific T‑cell‑redirecting antibody, as a second‑line monotherapy for relapsed/refractory multiple myeloma. The Phase III MajesTEC‑9 trial, enrolling 614 patients, demonstrated a 71 % reduction in...
Regulatory Pharmaceutical Fellowship Program
Several U.S. universities have launched two‑year medication safety fellowships that rotate participants through academic training, a major pharmaceutical company, and the FDA. Butler University offers two tracks with Regeneron and Eli Lilly, Purdue partners with AbbVie, and Rutgers collaborates with Pfizer....
Peptides in the Pipeline: How Companies Are Overcoming the Early-Stage Challenges
Peptide therapeutics are booming, with more than 2,000 candidates in discovery and pre‑clinical stages and projected global sales of $91.4 bn by 2025. Companies like Orocidin faced severe synthesis and purification hurdles for their QR‑01 peptide, but Syngene’s custom low‑loading resin...
Johns Hopkins Leads $24M Multinational Consortium to Find Hepatitis B Cure
Johns Hopkins Medicine is heading a five‑year, $24 million NIH‑funded Hepatitis B and HIV Cure Consortium that brings together research teams from the United States, Brazil, India, Senegal and Uganda. The first year will enroll 450 participants co‑infected with HIV and chronic...
Efgartigimod Effective in Treating Juvenile Myasthenia Gravis
A multicenter retrospective study of 17 Chinese patients with juvenile myasthenia gravis found that weekly efgartigimod 10 mg/kg for four weeks produced rapid and substantial clinical improvement. Clinically meaningful improvement was observed in 70.6% of patients by week 1 and 91.7% by...
BioNTech Co-Founders Launch New mRNA Company
BioNTech announced it will create an independent biotech company dedicated to next‑generation mRNA technologies. The spin‑out will be led by co‑founders Ugur Sahin and Özlem Türeci, who will assume management by the end of 2026. BioNTech will contribute core mRNA assets in...
CSL Breaks Ground on $1.5B Illinois Immunoglobulin Plant Expansion
CSL‑Behring broke ground on a $1.5 billion expansion of its Kankakee, Illinois manufacturing complex, slated to be operational by 2031. The project will add at least 300 pharmaceutical positions and roughly 800 construction jobs, with the state offering more than $200 million...
A Dose of Psilocybin Helps Smokers Quit in New Study
Researchers at Johns Hopkins found a single dose of psilocybin dramatically increased smoking cessation rates compared with nicotine patches. In a randomized trial of 82 smokers, 17 of 41 participants who received psilocybin remained abstinent after six months versus four...
First-of-Its-Kind Vaccine Protects Children From Deadly E. Coli Infections
Scientists announced ETVAX, the first oral vaccine that targets enterotoxigenic *E. coli* (ETEC) in children, after a large‑scale trial in The Gambia. The study involved 4,936 infants aged six to 18 months and demonstrated a 48% reduction in moderate‑to‑severe ETEC...
STAT+: Novo Nordisk Is Warned by the FDA for Failing to Report Side Effects Tied to GLP-1 Drugs
Novo Nordisk received an FDA warning letter on March 5, 2026 for failing to report suspected side effects of its GLP‑1 medicines. The violations were uncovered during a 2025 inspection of the company’s facilities and were described as “serious.” The agency warned...
US Vaccine Manufacturers Set to Lose as RFK Pushes for Weaker Vaccine Mandates
The Trump administration, led by Health Secretary Robert F. Kennedy Jr., plans to eliminate over a third of the CDC childhood vaccine schedule, targeting seven of eleven vaccines. The United States, which produces roughly 36% of global vaccine output, could...
Building for Decades: Dubai’s Long Game in Life Sciences
Dubai is positioning itself as a long‑term hub for healthcare and life sciences through two complementary free‑zone clusters – Dubai Healthcare City (DHCC) and Dubai Science Park (DSP). DHCC is undergoing a $1.3 bn expansion that will create six tightly linked...
FDA Sets Scope for Attempt To Reduce Manufacturing-Related Approval Rejections
The FDA has defined the scope of pre‑submission facility meetings to curb manufacturing‑related complete response letters that delay drug approvals. It agreed to cover prior production‑site inspections, novel process elements and supply‑chain node strategies, while rejecting topics such as alternative...
Desert to Discovery: Five Hot Biotechs in Arizona
Arizona's biotech sector is booming, with $3.7 billion invested over the past seven years and a growing pipeline of innovative companies. Nectero Therapeutics secured a $96 million Series D to develop a fast‑track, breakthrough‑designated endovascular treatment for abdominal aortic aneurysms, while Humabiologics obtained...
STAT+: Pharmalittle: We’re Reading About a U.S. Senator Probing FDA, BioNTech Founders Leaving, and Much More
BioNTech co‑founders Ugur Sahin and Özlem Türeci will leave the company by year‑end to launch an unnamed mRNA‑focused venture, with BioNTech retaining a minority stake and licensing key technology. The split allows BioNTech to concentrate on its late‑stage cancer pipeline...
China Is Going After the World's Most Expensive Drugs: Endpoints Signal
China is actively targeting ultra‑expensive gene therapies, challenging the notion that these treatments are immune to price competition. Recent regulatory approvals and centralized procurement initiatives have forced manufacturers to negotiate steep discounts on therapies that once commanded multi‑million‑dollar price tags....
FDA Widens Use of Leucovorin without New Trial Data
The U.S. Food and Drug Administration has approved the generic form of leucovorin, also known as folinic acid, for treating cerebral folate deficiency (CFD). The decision widens the drug’s label despite the absence of new clinical trial data, relying on...
Cell and Gene Therapy Manufacturing Market to Skyrocket to $146B by 2032
Credence Research forecasts the global cell and gene therapy manufacturing market to surge from $19.3 billion in 2024 to $146.2 billion by 2032. The compound annual growth rate is estimated at 28.8%, propelled by rising demand for advanced therapies, expanding commercialization, and...
Statement on Biomedical Countermeasures Initiative
BIOTECanada welcomes the launch of the National Research Council of Canada’s Biomedical Countermeasures Initiative, a key component of the federal Defence Industrial Strategy. The program is designed to strengthen Canada’s domestic capacity to develop and manufacture diagnostics, vaccines, and therapeutics...
Rising Amid Flurry of CAR T Deals, Stylus Proves Cell Therapy Is Not Dead
Stylus Medicine entered the cell‑therapy arena in May 2025 with an in‑vivo CAR‑T platform that delivers a lipid nanoparticle‑encapsulated recombinase to engineer T cells inside patients. The move comes after major pharma acquisitions—BMS buying Orbital Therapeutics for $1.5 billion and Gilead...
The Reputational Risk Hidden Inside Drug Pricing
Drug pricing is increasingly viewed as a reputation issue, not just a financial decision. Carreen Winters of MikeWorldWide argues that pricing choices shape public perception of a company's values, as illustrated by Novo Nordisk’s 50% cut to GLP‑1 drug prices....
India's Exports of Active Pharma Ingredients at Rs 41,500 Cr Surpassed Imports in FY25
India’s active pharmaceutical ingredient (API) market posted a landmark trade balance in FY25, with exports climbing to roughly Rs 41.5 billion while imports lagged at Rs 39.2 billion. The surplus marks the first time the sector has out‑exported its imports, signaling a maturing domestic...
Samsung Biologics, Eli Lilly to Establish Biotech Incubator in Incheon
Samsung Biologics and U.S. pharma leader Eli Lilly have signed an agreement to launch a biotech incubator in Incheon’s Songdo district. The facility will host Lilly’s Gateway Labs platform, offering lab space, equipment, funding and R&D collaboration to emerging biotech firms....
Dyne Plans Post-Prasad FDA Run as Duchenne Exon Skipper Sustains Benefit in Long Term Data
Dyne Therapeutics reported that its exon‑skipping candidate z‑rostudirsen sustained respiratory and cardiac benefits through 24 months in the Phase 1/2 DELIVER study for Duchenne muscular dystrophy. The therapy maintained forced vital capacity, circumferential strain and left‑ventricular ejection fraction improvements compared with...
Junshi Receives China’s NMPA Acceptance for Toripalimab Injection
Junshi Biosciences has received acceptance from China’s National Medical Products Administration for its subcutaneous toripalimab injection (JS001sc), covering 12 cancer indications. The filing marks the first domestic anti‑PD‑1 monoclonal antibody in a subcutaneous formulation to reach the marketing application stage....
Ascletis Announces Positive Topline Results From U.S. Phase II, 24-Week Study for Its Ultra-Long-Acting Subcutaneous Depot Formulations of Small Molecule...
Ascletis Pharma reported positive topline data from a U.S. Phase II, 24‑week study of its ultra‑long‑acting subcutaneous depot GLP‑1R agonist ASC30. The once‑monthly formulation A1 produced a placebo‑adjusted mean weight loss of 7.5% at week 16 and sustained a 5.8%...
BioNTech Co-Founders to Exit Company and Start a New One
Uğur Şahin and Özlem Türeci announced they will leave BioNTech by the end of 2026 to launch a new company focused on next‑generation mRNA technologies. BioNTech will concurrently narrow its portfolio, concentrating on late‑stage therapeutic candidates and its existing vaccine...
STAT+: Large Drugmakers Are Developing Fewer Antibiotics, Analysis Finds
A new analysis shows the world’s largest pharmaceutical firms cut antimicrobial development by 35% over the past five years, dropping from 92 to 60 candidates. Only five of the 39 pipeline projects aimed at WHO priority pathogens include pediatric formulations...
NewcelX and Eledon Partner for NCEL-101 Programme
NewcelX has entered a collaborative research agreement with Eledon Pharmaceuticals to advance its NCEL‑101 cell therapy for type‑1 diabetes. The partnership integrates NewcelX’s off‑the‑shelf islet replacement product with Eledon’s anti‑CD40L monoclonal antibody, tegoprubart, which has been used in over 100...
Why the FDA Is Embracing Old Math for New Drugs
The FDA released draft guidance encouraging the use of Bayesian statistics in drug and biologic clinical trials, aiming to shorten development timelines and lower costs. By allowing external data—known as priors—to be incorporated, the approach promises more efficient, adaptive studies,...
Single Pivotal Trials Demand Stronger Data and Risk Strategies
Following the FDA’s recent shift to require only one pivotal trial for new drug applications, sponsors now face heightened pressure to generate robust efficacy and safety data. Regulators expect a single, bullet‑proof study rather than two less conclusive trials, mirroring...
Rapport Therapeutics Partners with Tenacia Biotechnology to Advance RAP-219 in Greater China
Rapport Therapeutics has granted Tenacia Biotechnology exclusive rights to develop and commercialize its TARPγ8‑specific AMPA receptor negative allosteric modulator RAP‑219 in Greater China, covering indications such as focal onset seizures and bipolar mania. The agreement provides Rapport with a $20 million...
Mouse Brain Study Reveals Why Blockbuster Weight-Loss Drugs May Work Differently in Females and Males
Researchers at Icahn School of Medicine created the first sex‑specific atlas of GLP‑1 expression in the mouse brain using RNAscope, mapping the peptide across 25 nuclei. The atlas shows pronounced differences between females and males, especially in hindbrain nuclei of...
Nonprofit Aims To Modernize Manufacturing by Clearing Capital Hurdle
A nonprofit, the API Innovation Center (APIIC), is tackling the capital barrier that prevents U.S. drug makers from adopting continuous manufacturing. By pooling state, federal, philanthropic and private funds, APIIC installs equipment at manufacturers at no upfront cost, de‑risking the...
Other News to Note for March 10, 2026
Merck released phase III data from its Litespark‑011 trial, showing a potent HIF‑2α inhibitor that could reshape renal‑cell carcinoma therapy. Researchers at INSERM uncovered hypothalamic tanycytes as a primary mechanism for clearing pathological tau, opening new avenues for Alzheimer’s treatment....
Biontech SE (BNTX) Q4 2025 Earnings Call Transcript
BioNTech’s Q4 2024 earnings call highlighted a strategic pivot toward oncology, emphasizing the advancement of its bispecific antibody BNT327 and the recent acquisition of Biotheus to secure Chinese manufacturing and development capabilities. The company reported positive Phase 2 data for...
ADC Therapeutics SA (ADCT) Q4 2025 Earnings Call Transcript
ADC Therapeutics reported Q4 2025 product revenue of $15.8 million, down from $18 million a year earlier, while GAAP net loss narrowed to $41 million ($0.30 per share). Non‑GAAP operating expenses fell 12.1% to $45 million, driven by lower R&D spend. A $60 million private...
Legend Biotech Corp (LEGN) Q4 2025 Earnings Call Transcript
Biogen reported full‑year 2025 non‑GAAP diluted EPS of $15.28 and $9.9 billion in revenue, a modest 2% YoY increase. Growth‑product sales surged 19% to $3.3 billion, driven by VUMERITY, SKYCLARIS, ZERZUVE and CALSADI, while legacy MS revenues are projected to fall mid‑teens...
Hidden Blood Mutations Drive Severe Inflammatory Bowel Disease, but a New Treatment Target Is in Sight
Indiana University researchers linked clonal hematopoiesis of indeterminate potential (CHIP) to heightened severity of inflammatory bowel disease. Analysis of UK Biobank and All of Us data showed women with DNMT3A mutations and younger individuals with TET2 mutations face higher Crohn’s...
Gene Edit Makes Probiotic Safer for Immunocompromised Patients
An international team genetically deleted the ENA1 gene from Saccharomyces boulardii, a common probiotic yeast. In immunosuppressed mice, the ENA1‑deficient strain showed no mortality, raising survival from 30‑40% to 100% compared with wild‑type isolates. The edit also reduced osmotic stress...
Senate Dems Ask How Trump’s MFN Deals Mesh With GENEROUS Model
Senate Democrats have pressed major pharmaceutical companies to clarify the secretive most‑favored‑nation (MFN) pricing agreements they struck with the Trump administration in December. Lawmakers are questioning how those deals align with the Centers for Medicare & Medicaid Services’ new GENEROUS...
Roche’s Giredestrant Miss Refines Treatment Settings for Oral SERDs
Roche’s oral selective estrogen receptor degrader (SERD) giredestrant failed to meet its primary endpoint in a late‑stage Phase III trial, but the data revealed meaningful activity in specific patient subgroups, particularly those with ESR1 mutations. The miss prompted Roche to...