About CDER Small Business and Industry Assistance (SBIA)
The FDA’s Center for Drug Evaluation and Research (CDER) Small Business and Industry Assistance (SBIA) serves as a primary contact point for small pharmaceutical firms seeking regulatory guidance. SBIA provides direct phone and email support, a dedicated web portal, and a suite of training resources including on‑demand webinars and podcasts. Its mission is to give industry stakeholders immediate access to education and updates, helping streamline drug development and approval. The office also defines a small business as any entity with fewer than 500 employees, affiliates included.
Harbour BioMed Gains FDA Clearance for First-in-Human Study of B7H4xCD3 Bispecific Antibody HBM7004
Harbour BioMed announced FDA IND clearance to launch a Phase I first‑in‑human study of its bispecific antibody HBM7004, which targets B7H4 and CD3 in advanced solid tumors. The trial will assess safety, tolerability, pharmacokinetics and early anti‑tumor activity across multiple cancer...
FDA Rolls Out 1-Day Assessment Pilot in Bid to Refocus Inspection Resources
The FDA announced a pilot program that conducts one‑day inspectional assessments to better allocate its inspection resources. The initiative aims to streamline oversight by focusing on higher‑risk facilities while providing clearer expectations for manufacturers. Cooley’s regulatory partner Sonia Nath said...
Daiichi Sankyo Posts 'Extraordinary Loss' Of Nearly $1B
Daiichi Sankyo announced it is scrapping its planned antibody‑drug‑conjugate (ADC) manufacturing line, a move that triggered an extraordinary loss of 149.4 billion Japanese yen (approximately $950 million). The loss reflects a write‑down of capital expenditures and R&D investments tied to the abandoned...
Biosimilar Ramp-Up to Drive Growth for Biocon in FY27
Biocon is pivoting from a heavy‑investment phase to a profit‑driven model, banking on new biosimilar launches and scale‑up of recent products to fuel FY27 growth. Biosimilars already accounted for 60% of FY26 revenue, delivering 16% year‑on‑year top‑line growth and a...
Some Gene Therapies No Longer Require Clinical Trials, Thanks to New FDA Rule. Is This Safe, and Who Will It...
The FDA has introduced a "plausible mechanism pathway" that lets developers market experimental gene‑editing therapies for rare, monogenic disorders without completing traditional large‑scale clinical trials. The rule relies on prior safety data for the delivery platform and permits customization of...
Can Existing Flu Shots Help Protect Against Bird Flu?
Researchers from National Taiwan University and the University of South Florida analyzed 35 ferret studies spanning two decades and found that seasonal influenza vaccines containing the neuraminidase N1 protein reduced H5N1‑related mortality by roughly 73%. By contrast, vaccines without N1...
Japan’s FY26 Price Revision Expands G1 Repricing and Drives Price Reduction
Japan’s FY26 National Health Insurance drug price revision took effect in April 2026, lowering average prices by 4.02% across about 15,800 products. The revision expands the G1 repricing rule to all off‑patent medicines and biologics with biosimilar competition, driving price...
Medical Marijuana Removed From Schedule I – Moving Closer to Broadcast and Online Advertising but Concerns Still Remain
The Justice Department and DEA moved FDA‑approved medical marijuana and state‑licensed medical cannabis from Schedule I to Schedule III, creating a limited federal pathway for distribution. Recreational cannabis remains Schedule I, so its advertising stays prohibited. The change allows federally registered dispensaries to...
Bayer Announces Positive Data for Investigational PET Radiotracer
On May 7, 2026 Bayer announced that its investigational PET radiotracer I‑124 evuzamitide achieved positive topline results in the Phase III REVEAL study, meeting primary sensitivity and specificity endpoints for diagnosing cardiac amyloidosis. The multicenter trial evaluated 170 adults at 19 U.S....
A Monocyte‐Targeted Nanoplatform for Phagocytosis Activation and Ferroptosis Inhibition in Intracerebral Hemorrhage
Researchers have engineered a monocyte‑targeted nanoplatform (mPDA@DFO‑CpG‑N1) to accelerate hematoma clearance after intracerebral hemorrhage (ICH). The system combines a high‑affinity aptamer for selective monocyte delivery, a TLR9 agonist that overrides CD47‑SIRPα inhibition, and the iron chelator deferoxamine to block ferroptosis....
FDA Delays Leqembi Decision; Artiva Raises $300M; Pharvaris Prices Offering
The FDA has postponed its decision on the subcutaneous formulation of Leqembi, moving the target date to August 24, delaying a potential launch for Eisai and Biogen’s early‑Alzheimer’s therapy. The shift adds uncertainty to the Alzheimer market, where Leqembi could become...
AI-Guided Labs Are Approaching Full Autonomy
Ginkgo Bioworks is building fully autonomous biopharma labs that combine 70 robots, 90 lab devices and AI‑driven software in an 18,000‑square‑foot Boston facility. Scientists can submit dozens of unique protocols each day, which the system executes without human intervention. In...
ParcelBio Unveils Programmable mRNA Platform Backed by $13M Financing
ParcelBio announced a $13 million seed round led by Breyer Capital, with participation from General Catalyst, Y Combinator and other investors. The funding will accelerate its proprietary Amplified and Prolonged Expression mRNA (APEXm™) platform, which claims to deliver markedly higher and longer‑lasting...
Merck Advances Scalable Manufacturing for Oral PCSK9 Therapy
Merck has published a landmark study in Science describing a scalable biocatalytic synthesis platform for its investigational oral PCSK9 inhibitor, enlicitide decanoate. The enzyme‑driven process enables selective peptide fragment formation, coupling, macrocyclization and uses crystallization‑based purification, aiming to reduce reliance...
Communicating Benefit and Risk Information
The FDA’s Division of Drug Information highlighted its dual‑track approach to benefit‑risk communication: direct patient‑oriented counseling and sponsor‑directed guidance for manufacturers. Since 1999, the agency has mandated Medication Guides for drugs with serious adverse‑effect potential, and it enforces balanced risk...
Drug Promotion
The FDA oversees all promotional materials for prescription drugs, ranging from TV spots to website content, while the FTC handles over‑the‑counter products. Companies cannot obtain pre‑approval, but must submit their ads to the agency at the time they go public,...
STAT+: Pharmalittle: We’re Reading About an FDA Gender Rule Change, Its Program for One-Day Inspections, and More
The U.S. Food and Drug Administration announced a proposed rule to strip references to “gender” from its regulations, aligning with a 2025 Trump administration executive order that stresses biological sex. While the agency claims the change won’t affect industry practice,...
Capricor Sues Partner Nippon Shinyaku in DMD Therapy Spat
Capricor Therapeutics has filed a lawsuit against its Japanese partner Nippon Shinyaku and its U.S. subsidiary NS Pharma, alleging a fundamental pricing flaw in their distribution agreement for the Duchenne muscular dystrophy (DMD) cell therapy deramiocel. The biotech claims the...
ASGCT Honors Mohamed Abou‑el‑Enein as Outstanding New Investigator
Mohamed Abou‑el‑Enein, MD, PhD, received the American Society of Gene and Cell Therapy’s 2026 Outstanding New Investigator Award and his lab earned the Best of Molecular Therapy Award. His team’s high‑dimensional spectral flow cytometry platform maps CAR‑T cell states, pinpointing...
Clostridioides Difficile Infection: Developing Drugs for Treatment, Reduction of Recurrence, and Prevention
The U.S. Food and Drug Administration has issued a final Level 1 guidance titled “Clostridioides difficile Infection: Developing Drugs for Treatment, Reduction of Recurrence, or Prevention.” The document replaces the October 2022 draft and provides detailed recommendations for clinical trial design, endpoints,...
Pulmonary Tuberculosis: Developing Drugs for Treatment
The FDA has issued a final Level 1 guidance titled “Pulmonary Tuberculosis: Developing Drugs for Treatment,” replacing the draft released in December 2022. The guidance, docket FDA‑2013‑D‑1319, outlines clinical development expectations for new antibacterial agents targeting pulmonary TB. It provides sponsors with detailed...
Postapproval Pregnancy Safety Studies
The FDA has released its final Level 1 guidance on post‑approval pregnancy safety studies, updating the 2019 draft to provide detailed recommendations for designing investigations of drug and biologic exposure in pregnant women. The guidance outlines methodological standards, data sources, and...
What Is PDUFA—And Why Does It Matter for Biotech Innovators, FDA & Patients?
Congress will reauthorize the Prescription Drug User Fee Act (PDUFA) next year, renewing the FDA’s authority to collect fees from biotech firms for drug reviews. The user‑fee model supplies a stable budget that underpins faster, more predictable approval timelines—about ten...
The US FDA Grants IND Clearance to Harbour BioMed’s HBM7004 to Initiate P-I Trial in Advanced Solid Tumors
The U.S. Food and Drug Administration has granted IND clearance for Harbour BioMed’s bispecific antibody HBM7004, allowing a Phase I trial in patients with advanced solid tumors. HBM7004 links the tumor‑associated antigen B7H4 to CD3, redirecting T‑cells to the tumor...
Eisai, Biogen Face Delay to Subcutaneous Leqembi
The FDA has extended its review of Eisai and Biogen’s subcutaneous Leqembi Iqlik, moving the decision on its use as a starting dose to August 24. The agency requested a major amendment to the marketing application but has not raised safety...
Gilead Sets Blockbuster Bar for Yeztugo’s First Full Year on the Market
Gilead announced a $1 billion sales target for its twice‑yearly PrEP injection Yeztugo in 2026, up from a prior $800 million outlook. The drug generated $166 million in Q1, a 72% increase from the previous quarter, and 95% of U.S. patients enjoy $0...
Ascendis Retreats to Rare Endocrinology Wheelhouse as Cancer Plans Falter
Ascendis Pharma announced it will halt development of onvapegleukin alfa, its IL‑2 β/γ oncology candidate, after a Phase 1/2 trial showed modest survival benefit in platinum‑resistant ovarian cancer. The company cited a strategic mismatch and will instead concentrate on its rare‑endocrinology franchise,...
Capricor Sues Nippon Shinyaku over Duchenne Drug 'Pricing Flaw' And Launch Prep
Capricorn Therapeutics has filed a lawsuit against Japan’s Nippon Shinyaku, alleging a breach of their licensing agreement for the Duchenne muscular dystrophy cell‑therapy candidate deramio‑cel. The complaint centers on a pricing flaw that Capricorn says Nippon Shinyaku misrepresented during launch...
Streamline Data Capture and Automate EHR-to-EDC Data Transfer
Paradigm Health introduced eSource Casebook, an EHR‑integrated platform that extracts both structured and unstructured clinical data, auto‑populates electronic case report forms, and streams the information directly to sponsor EDC systems. The tool tackles the industry‑wide surge in manual data entry,...
MEDSIR Reports PHERGain and PHERGain-2 Trial Results for Breast Cancer
MEDSIR presented Phase II data from the PHERGain and PHERGain‑2 trials, exploring chemotherapy‑free strategies for early HER2‑positive breast cancer. PHERGain showed that PET‑guided use of trastuzumab and pertuzumab allowed roughly 30% of patients to omit chemotherapy while achieving nearly 90% five‑year...
Manufacturing Hangover Drives Daiichi Sankyo Into the Red
Daiichi Sankyo announced a fiscal‑year loss of ¥149.4 billion (about $950 million) despite a 12.5% revenue increase to ¥2,123 billion ($13.4 billion). The shortfall stems from costly third‑party manufacturing contracts for its antibody‑drug conjugates (ADCs) and an impairment of ¥19.3 billion ($123 million) after scrapping an...
Venom and Hot Peppers Offer a Key to Killing Resistant Bacteria
Researchers at Mexico’s UNAM have created three new antibiotics from scorpion venom and habanero pepper compounds. Two benzoquinone molecules from Diplocentrus melici venom show activity against tuberculosis, Staphylococcus aureus and Acinetobacter baumannii, while a defensin peptide from Capsicum chinense targets...
ALX Oncology Presents P-I/II Trial Data on Evorpacept Combination in Metastatic Breast Cancer (mBC) at ESMO Breast Cancer’26
ALX Oncology reported exploratory Phase Ib/II data on evorpacept combined with Ziihera in 24 heavily pre‑treated HER2‑positive metastatic breast cancer patients, all of whom had previously received Enhertu. Overall, the regimen achieved a 33% confirmed overall response rate (cORR) with...
Beacon Therapeutics Reports P-II (DAWN) Trial Data on Laru-Zova in X-Linked Retinitis Pigmentosa (XLRP)
Beacon Therapeutics presented 12‑month data from its Phase‑2 DAWN trial of laruparetigene zovaparvovec (laru‑zova) in patients with X‑linked retinitis pigmentosa (XLRP) who previously received an AAV‑RPGR gene therapy. The study showed sustained gains in low‑luminance visual acuity (LLVA) and microperimetry mean...
Semaglutide Race: Torrent Grabs 38% Market Share
Torrent Pharma has seized a 38% share of India’s generic semaglutide market within a month of launch, generating about ₹17 crore ($2 million) in April sales. Generic semaglutide revenue reached ₹44 crore ($5.3 million), while total market sales—including Novo Nordisk’s innovator—hit roughly ₹89 crore ($10.7 million)....
Screening for Photoreceptor Survival
Researchers used human retinal organoids to screen compounds that affect cone survival under glucose starvation. They identified two kinase inhibitors, CS‑KI‑1 and CS‑KI‑2, targeting CK1 and MAPK11, that protected cones and rods in vitro and in a mouse model of...
Amplia Therapeutics Launches New Narmafotinib Ovarian Cancer Study with ANZGOG
Amplia Therapeutics announced the launch of the PRROSE investigator‑initiated trial, testing its FGFR inhibitor narfotininb together with carboplatin and paclitaxel in high‑grade serous ovarian cancer patients who responded poorly to initial platinum therapy. The study will enroll 15‑20 participants, prioritize...
Spermidine Halts Liver Fibrosis by Cell Signal Remodeling
Researchers have demonstrated that spermidine, a naturally occurring polyamine, can halt the progression of liver fibrosis by reprogramming cellular signaling pathways. In mouse models, spermidine treatment reduced collagen deposition and restored normal liver architecture within eight weeks. The study identified...
Two Mechanisms Vie to Deliver First Hypoxic Ischemic Encephalopathy Drug
The article is BioCentury’s cookie policy, outlining five cookie categories—strictly necessary, functional, marketing, advertising, and analytics—and describing their purposes for site operation, personalization, outreach, and data collection. It explains how each type works, the data it handles, and the impact...
Astellas Touts Data From Early Test of Stem Cell-Derived Eye Therapy
Astellas Pharma announced early-stage data from its stem cell‑derived retinal therapy, aimed at treating age‑related macular degeneration (AMD). In a small cohort receiving the highest dose, patients showed statistically significant gains in best‑corrected visual acuity and no serious safety signals....
Novel Nanoparticle Therapy Using Manganese Could Improve Cancer Treatment
Researchers at the University of Michigan and MD Anderson have engineered a manganese‑based nanoparticle named CRYSTAL that activates the cGAS‑STING immune pathway without provoking systemic inflammation. Preclinical studies across several tumor models, including advanced triple‑negative breast cancer, demonstrated robust, sustained...
G-Link CAR-T Delivery Platform Showcased at ASGCT
Vyriad unveiled its G‑Link CAR‑T delivery platform at the ASGCT meeting, showcasing a modular protein adapter that repurposes existing lentiviral vectors for in‑vivo use. The technology promises to cut development timelines by eliminating extensive vector redesign and to boost transduction...
FDA Approval of DOR/ISL Expands HIV Treatment Options Beyond INSTIs: Amy Colson, MD, MPH
The FDA has approved Idvysno, a two‑drug regimen of doravirine and islatravir (DOR/ISL), marking the first HIV therapy that omits both integrase strand transfer inhibitors (INSTIs) and tenofovir. Clinical trials showed non‑inferior viral suppression at 48 weeks compared with standard...
Rare Disease News, Events & Reports
The FDA’s Center for Drug Evaluation and Research (CDER) continues to expand its Rare Disease Innovation Hub and Accelerating Rare Disease Cures (ARC) program through a steady stream of workshops, public meetings, and reports from 2022 to 2026. Recent highlights...
With Legal Briefs in, Supreme Court Weighs Telehealth Access for the Abortion Pill
The Supreme Court issued a one‑week stay on the 5th Circuit’s May 1 ruling that would have ended telemedicine prescribing of the abortion pill mifepristone nationwide. The pause keeps the FDA’s 2023 rule—allowing mail‑order and pharmacy dispensing—alive until at least May 11,...
The BioPharm Brief: Breakthrough Signals in Cancer, Duchenne, and RNA Medicine
Aptevo Therapeutics reported an 87% clinical benefit rate and 81% remission in its Phase 1b AML RAINIER study, combining mipletamig with venetoclax and azacitidine. Entrada Therapeutics disclosed positive Phase 1/2 data for ENTR‑601‑44, showing functional gains and increased dystrophin in Duchenne patients...
Gene Therapy Restores Walking After Paralysis
Researchers at the University Hospital Cologne used a designer cytokine, hyper‑interleukin‑6 (hIL‑6), delivered via an AAV2 viral vector into the motor cortex of mice with spinal‑cord contusions. The protein traveled transneuronally to brainstem serotonergic neurons, prompting intact fibers to sprout...
CMS to Provide Medicare Part D Beneficiaries with $50 Monthly Access to Certain GLP-1 Medications
The Centers for Medicare & Medicaid Services announced a short‑term demonstration called the Medicare GLP‑1 Bridge, offering eligible Part D beneficiaries access to selected GLP‑1 drugs for $50 per month. The benefit starts July 1 2026 and runs through Dec 31 2027. The program is...
DUET Trial: Low-Sodium Oxybate Significantly Consolidates Nighttime Sleep Architecture in Narcolepsy
The phase‑4 DUET trial showed that low‑sodium oxybate (LXB, Xywav) markedly consolidates nighttime sleep in narcolepsy type 1 and type 2 patients. Objective polysomnography recorded a 45‑minute increase in deep N3 sleep, 13 fewer stage transitions and three fewer awakenings per night....