Know What's Happening in Pharma
Know What's Happening in Pharma
FDA approves KRESLADI, first CIRM‑funded gene‑editing therapy for LAD‑I
The FDA has granted approval to KRESLADI, a gene‑editing therapy from Rocket Pharmaceuticals that treats severe leukocyte adhesion deficiency‑I in children without a matching bone‑marrow donor. This marks the first FDA‑approved product directly funded by California’s Institute for Regenerative Medicine (CIRM), highlighting the agency’s impact on translational research.
Also developing:
By the numbers: Lupin Limited acquires VISUfarma from GHO Capital
Buzz Health President Joseph Kleiman says TrumpRx is advancing drug‑price transparency by giving consumers early visibility into prescription costs. He notes that real‑time pricing can help patients compare options but also warns that prices are dynamic and may change before a fill. Operationally, Kleiman highlights consistency and drug availability as major hurdles, especially when high demand creates shortages. The interview underscores both the consumer benefits and the logistical complexities of scaling a transparent pricing platform.
Otsuka America became the first major pharmaceutical company to back psychedelic‑inspired medicines in 2020 through a partnership with COMPASS Pathways, and deepened that commitment by acquiring Mindset in 2023. The company is applying its long‑standing CNS expertise to develop serotonergic...
Merck Animal Health announced that the U.S. FDA has approved Numelvi (atinvicitinib) for controlling pruritus linked to allergic dermatitis in dogs six months and older, with commercial availability slated for spring 2026. Numelvi is a second‑generation JAK inhibitor that demonstrates...
The FDA has rejected Regenxbio’s gene therapy for mucopolysaccharidosis type 2, citing a need for additional data that could postpone approval by years. This decision adds to at least four other recent rejections of cell or gene therapies targeting deadly rare...
Mahzi Therapeutics has administered the first dose of its investigational gene therapy MZ-1866 in the Phase I/II UNITE study for Pitt‑Hopkins syndrome. The open‑label, multicentre trial will enroll roughly 12 genetically confirmed patients across sites in Spain, Israel and the...
Women’s HealthX (WoHX) will convene 750 pharma, biotech, hospital, insurer, startup and government leaders in Boston on December 3‑4, 2026. The two‑day summit aims to close the sex‑difference data gap by showcasing evidence‑based technologies, AI tools and digital therapeutics across the...
The Trump administration launched TrumpRx, a website aggregating manufacturer discounts on 43 brand‑name drugs and touting presidential credit for lower patient costs. Early analysis shows that 20 of the listed products already have cheaper generic versions, and the portal deliberately...
argenx announced topline Phase III ADAPT OCULUS data for Vyvgart (efgartigimod alfa + hyaluronidase‑qvfc) in adults with ocular myasthenia gravis. The trial met its primary endpoint, delivering a 4.04‑point improvement in Myasthenia Impairment Index PRO ocular scores versus 1.99 points for placebo at...
The Central Drugs Standard Control Organization (CDSCO) will issue No Objection Certificates (NOC) for drug‑sample testing immediately upon receipt of applications, eliminating the prior detailed specification review. Companies can begin laboratory testing at designated Indian Pharmacopoeia Commission and other central...
The Fifth Circuit rejected the DEA’s long‑standing reading of the pharmacist’s “corresponding responsibility” and “usual course of professional practice” regulations, ruling that liability requires proof a prescription was invalid when issued and that the pharmacist actually knew it was invalid....
Emergent BioSolutions reported a successful completion of its stabilization phase, cutting net debt by $156 million and improving leverage to 3.3x adjusted EBITDA. Adjusted EBITDA surged to $183 million, a $205 million swing from the prior year, while operating cash...
Ascendis (SNDX) reported €720 million total 2025 revenue, driven by €477 million from Yorvipath and €206 million from Skytrofa. Q4 operating profit hit €10 million and cash flow reached €73 million, leaving €616 million in cash at year‑end. Management guided 2026 operating cash flow of roughly...
Researchers injected acetate, a common metabolic by‑product, into mice and tested long‑term memory using spatial and object‑recognition tasks. Female mice that received acetate showed significantly better performance 24 hours later, while male mice displayed little to no improvement. The memory...
Amphastar Pharmaceuticals reported full‑year 2025 net revenues of $719.9 million, a modest 2% decline, while its flagship product Baqsimi grew 12% to $185.4 million. The company secured FDA approval for ipratropium bromide HFA (AMP‑007), gaining 180‑day generic exclusivity, and announced a major...
Pacira BioSciences reported a record $726 million in total 2025 revenue and an all‑time high non‑GAAP gross margin of 80%, driven by growth in its flagship product EXPAREL. EXPAREL sales rose 7% year‑over‑year to $155.8 million, though the increase was partially offset...
Akebia Therapeutics reported 2025 total revenue of $236.2 million, a 47% increase driven by the commercial launch of Vafseo and higher Auryxia sales. Net product revenue reached $227 million, with Vafseo contributing $45.8 million and gaining prescribing access for 290,000 dialysis patients. The...
OPKO Health reported a $369 million cash position for Q4 2025, driven by asset sales, BARDA funding and partnership payments. The company repurchased 34.6 million shares for $47 million and allocated $109 million to stock and convertible note buybacks. Diagnostics revenue dropped to $71.1 million, but...
PolyPid announced successful completion of the Phase III SHIELD II trial, with D‑PLEX100 achieving its primary and key secondary endpoints and demonstrating a meaningful reduction in surgical site infections. The FDA provided written support for a rolling NDA review, and the company...
Recent pre‑clinical work has mapped the synaptic changes that underlie benzodiazepine tolerance. Chronic exposure uncouples GABA(A) receptors from their benzodiazepine binding site, down‑regulates α1‑subunit mRNA, and reshapes receptor trafficking through auxiliary proteins such as Shisa7. These alterations weaken inhibitory signalling,...
Anika Therapeutics reported total revenue of $27.8 million for the quarter, a 6% year‑over‑year decline driven primarily by lower pricing in its OEM osteoarthritis pain‑management channel. Commercial channel revenue rose 22% to $12 million, powered by a 25% surge in...
Ligand Pharmaceuticals reported a breakout 2025, delivering $268 million total GAAP revenue and $240 million core revenue, driven by a 48% surge in royalty income. Adjusted earnings per share rose 42% to $8.13, surpassing guidance by more than 30%, while cash and...
MannKind reported a record Q4 2024 with $77 million in revenue and a full‑year total of $286 million, a 43% increase year‑over‑year. Afrezza sales grew 17% to $64 million, and the company announced a pediatric filing for the inhaled insulin in the first...
Rhythm Pharmaceuticals reported a strong Q4 2025, with product revenue rising to $57.3 million—a 12% sequential increase and 37% year‑over‑year growth. Full‑year revenue jumped 50% to $194.8 million, driven by expanding U.S. sales and broader international access for IMCIVREE. The company highlighted...
Recent neuroimaging and tract‑tracing studies have identified a nuclei‑specific fronto‑amygdala pathway that links distinct amygdala subregions with ventromedial prefrontal cortex. The work maps serotonin, norepinephrine and dopamine receptor densities across these nuclei, revealing divergent pharmacological profiles for SSRIs and SNRIs....
Organogenesis Holdings posted a record fourth‑quarter 2025, delivering $225.1 million in net product revenue, a 78% year‑over‑year increase driven largely by an 83% surge in advanced wound‑care sales. Gross profit rose to 78% of revenue and GAAP operating income jumped 519%...
Researchers have engineered ligand‑conjugated mRNA‑lipid nanoparticles that home specifically to the endometrium, delivering therapeutic mRNA directly to uterine tissue. In a murine model of endometrial injury, the targeted formulation restored embryo implantation rates to near‑normal levels. Safety profiling showed reduced...
Definium Therapeutics reported that its lead psychedelic candidate DT120 ODT has fully enrolled the EMERGE Phase III trial for major depressive disorder, with top‑line results expected in late Q2 2026. The VOYAGE Phase III study for generalized anxiety disorder is...
BioCryst Pharmaceuticals reported a 37% year‑over‑year rise in Orladeyo revenue to $159.1 million in Q3 and lifted its 2025 sales guidance to $590‑$600 million. The company completed the divestiture of its European business, repaid a $200 million term loan and entered Q3 with...
Puma Biotechnology reported total 2025 revenue of $75.5 million, driven by $59.9 million net product revenue from its HER2‑positive breast cancer drug NERLYNX and a surge in royalty income to $15.6 million after a large shipment to a Chinese partner. Ex‑U.S. inventory rose...
Heron Therapeutics reported full‑year 2025 net product sales of $155 million and adjusted EBITDA of $14.7 million, surpassing its guidance. Acute Care drove growth, with ZYNRELEF revenue up 48% YoY and the franchise expanding 57% overall, helped by the CrossLink IGNITE incentive program,...
Kymera Therapeutics reported Q4 2025 revenue of $2.8 million, entirely from its Gilead collaboration, while maintaining a cash balance of $978.7 million that extends runway into the second half of 2028. Adjusted R&D and G&A expenses fell 7% and 3% quarter‑over‑quarter, respectively....
Intellia Therapeutics announced that the FDA has placed a clinical hold on its nex‑z MAGNITUDE and MAGNITUDE‑2 Phase III trials after a patient death and liver enzyme abnormalities. The company is implementing intensified safety monitoring and data reviews while awaiting regulatory...
Aktis Oncology announced that its investigational radiopharmaceutical AKY-1189 has earned FDA Fast Track designation. The drug targets Nectin‑4, present in 80‑90 % of urothelial cancers, and delivers the alpha‑emitter actinium‑225 directly to tumors. AKY-1189 is in a Phase 1b trial covering urothelial,...
Rare Disease Day marked its 11th anniversary, highlighting the stark disparity between the 8,000 known rare‑disease genes and the under 500 approved therapies. Hosted by the Broad Institute’s Ladders to Cures Accelerator and the Termeer Institute, the event featured leading...
A new Nature study led by ETH Zurich researchers sequenced the genomes of 645 bacteria and archaea from over 800 coral samples, revealing that more than 99% of these reef‑dwelling microbes were previously unknown. The analysis showed that each coral...
Long‑acting cabotegravir/rilpivirine (CAB+RPV LA) demonstrated strong virologic control and high patient preference in both treatment‑naïve and treatment‑experienced cohorts presented at CROI 2026. In the VOLITION trial, 85% of ART‑naïve adults switched early from DTG/3TC to a q2‑month injectable, achieving 95% overall suppression...
Brazilian researchers have shown that an iron‑based compound, ferroin, encapsulated in lipid nanoparticles, completely eradicated Mycobacterium tuberculosis from mouse lungs after a 30‑day course. The formulation, LNP@FEP, stabilizes the drug, enhances the activity of existing antibiotics, and targets bacterial cell‑wall synthesis....
2026’s leading women CEOs in pharma and biotech are steering some of the industry’s biggest strategic moves, from multi‑billion‑dollar investments to landmark FDA approvals. The list includes Julie Kim’s upcoming Takeda leadership, Emma Walmsley’s transition at GSK, Reshma Kewalramani’s growth...
The FDA has removed several accelerated approvals for non‑malignant hematological, neurological, and other disorder indications after post‑marketing requirements were withdrawn. These withdrawn indications are not reflected in official listings until a Federal Register notice or label update is issued. The...
Dotinurad (FYU‑981), marketed as Urece®, is a URAT1 inhibitor approved for gout and hyperuricemia in Japan and China. The drug was chemically refined from the older uricosuric benzbromarone to retain potency while eliminating rare hepatotoxic events. Crystalys Therapeutics is now...
The FDA’s ongoing accelerated‑approval list details 30+ drugs for non‑malignant hematologic, neurologic and other rare disorders that remain contingent on post‑marketing requirements (PMRs). Each entry cites the indication, approval date, specific confirmatory trial design, and a projected completion date ranging...
The FDA’s Accelerated Approval Program permits earlier market entry for drugs treating serious conditions by relying on surrogate endpoints that predict clinical benefit. Companies must later complete confirmatory trials; positive results convert the approval to traditional status, while negative outcomes...
Apheris has launched the ADMET Network, a federated learning platform that lets pharmaceutical companies jointly train ADMET prediction models without exposing raw data. Five founding members—Lundbeck, Orion Pharma, Recursion, Servier and an undisclosed partner—have each contributed roughly 80% of their...
Psychedelic tape still trades on vibes > policy. Surgeon General nominee Casey Means says she “would not recommend” psilocybin to the American people today. Yes, she nods to “exciting work” in PTSD and veterans. But that is a far cry from...
The combination of GLP-1 drugs (such as Ozempic) and healthy lifestyle factors was associated with less major adverse cardiovascular events in ~100,000 people with T2D @TheLancetEndo https://t.co/phNzEBy7zY https://t.co/DDVi8uedRP
The Xtalks Life Science Podcast’s Episode 244 spotlights a persistent gap in epilepsy care for pregnant women, noting that fewer than 5% of clinical trials include this population. Andrea Wilkinson, UCB’s Global Head of Patient Engagement & Advocacy, discusses new...
An experimental therapy for a rare blood cancer was on the path toward approval by the Food and Drug Administration last year — with internal reviewers recommending it be cleared — before the agency rejected the drug last month, @adamfeuerstein...
Interesting read: the thread and the story. A drug that was approved in Europe in 2022, which looked like it was on track for #FDA approval, instead is rejected. What changed at FDA? 🤔
The Federal Register notice for the (now) March #ACIP meeting is now up & it's ... puzzling. Possible vote on Covid vaccine injury? ACIP can recommend changes to the way a vaccine is used, for safety reasons. But vaccine injuries aren't...
$LTRN is a live experiment in our thesis: AI doesn’t need to invent new drugs. It needs to improve probability of success in humans. If RADR consistently enriches responders, that’s durable value. If not, platform premium disappears. Listen to the podcast here: https://t.co/ZAg5btpcIK
