Susan Haigney

In regulatory news in June 2026, the US FDA accepted the first in silico drug development tool as part of its ISTAND program, approved a variety of drugs that included treatments for rare conditions, and issued guidance on the acceleration of cell and gene therapy.

On June 3, 2026, the agency accepted a letter of intent for an 

 into its Innovative Science and Technology Approaches for New Drugs (ISTAND) DDT Qualification Program.

 The DDT is an artificial intelligence-driven digital liver model that predicts drug induced liver injury and will help reduce the use of animals in a nonclinical space and improve predictive toxicology in humans.

 posted June 8, Dr. Iilun Murphy, director Office of Generic Drugs, Center for Drug Evaluation and Research (CDER), and Dr. Mike Kopcha, director Office of Pharmaceutical Quality, CDER, provided insight into the agency’s approach to generic drug oversight.

 Murphy and Kocha acknowledged that the manufacturing of generic drugs is complex and encouraged manufacturers to adopt mature quality management practices beyond what is required by current good manufacturing practices. They also restated the FDA’s support for onshore manufacturing.

over-the-counter (OTC) intranasal naloxone product

, Rextovy, was approved by the FDA on June 16, 2026 for the emergency treatment of opioid overdose.

 The action is part of FDA’s priority to reduce opioid deaths by broadening access to these types of treatments that rapidly reverse the effects of opioid overdose.

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June saw regulators look back at their achievements, take stock of drug shortages, and advance treatments for rare diseases.

Regulatory Actions for June 2026

Regulatory Roundup June 2026

"The FDA’s approval of another OTC naloxone nasal spray is an important step in expanding access to a life-saving medication. At the same time, device format and manufacturability remain critical considerations. For customers developing nasal rescue products, early evaluation of filling approach, device compatibility, and supply-chain strategy will be essential," Deborah Smook, VP of Marketing & Business Development for TurboFil Packaging Machines, told PharmTech.

On June 15, the FDA approved teplizumab for a new indication to delay the decline of insulin production in children aged 8 to 17 with stage 3 type 1 diabetes. It’s the first treatment of the indication. Teplizumab was previously approved to delay stage 3 type 1 diabetes in adults and in children with stage 2 type 1 diabetes. The approval was granted through FDA’s accelerated approval pathway.

Cancer treatment development continues to be a priority for industry and regulators. On June 12, the FDA approved belzutifan, an adjuvant treatment for adults with renal cell carcinoma with a clear cell component.

 Also on June 12, the agency approved AstraZeneca’s Truqap in combination with abiraterone and prednisone for adults with metastatic androgen pathway modulation-naïve or -sensitive prostate cancer that is PTEN-deficient as detected by an FDA-authorized test.

On June 24, 2026, Pfizer and FDA announced the approval of a maintenance treatment for adults with hormone receptor-positive (HR+), human epidermal growth factor receptor 2-positive (HER2+) locally advanced or metastatic breast cancer (MBC).

Accelerating Therapy Development and Approval

The FDA has been making moves in supporting innovation in drug development. On June 22, 2026, the agency announced its plans to accelerate and modernize clinical research across the full continuum of drug development. Some of its efforts include a proposed Expedited Investigational New Drug (IND) Pilot Program that collaborates with research institutions to shorten the drug identification and approval time, a Phase 1 IND Navigator webpage, Phase 1 IND CMC wepage, and more.

Rare diseases and unmet needs are another area FDA has been recently focused on. In an 

 blog post, Karim Mikhail, acting director, Center for Biologics Evaluation and Research, Mike Davis, Acting Director, Center for Drug Evaluation and Research, and Amy Comstock Rick, director, Rare Disease Innovation Hub,detailed how the agency is committed to the development of treatments for rare diseases and how its decisions are based on rigorous science and expertise. They also talked about how the perspective of patients is critical to development.

European Medicines Agency (EMA) announced that the agency’s

 Emergency Task Force is discussing clinical trial designs and medical countermeasures to the Ebola outbreak in the Democratic Republic of the Congo and Uganda with the African Medicines Agencyand its national regulatory authorities.

 The organizations are leveraging expertise from the World Health Organization-AFRO African Vaccines Regulatory Forum to address the Ebola outbreak. The collaboration will support “efficient, coordinated and timely regulatory responses to the outbreak.”

 in 2025, according to a report the agency issued on June 11, 2026.

The report also detailed the agency’s advances in biosimilar policy, medicines availability, and the integration of artificial intelligence (AI) into agency operations.

On June 12, EMA’s Pharmacovigilance Risk Assessment Committee (PRAC) provided highlights from its June meeting, specifically PRAC finished it review of valproate and concluded that the data were inconsistent on the development of neurodevelopmental disorders in children born to men treated with valproate.

Other EMA meeting highlights include notes from EMA’s Management Board meeting, which consisted of a review of the agency’s work on the Ebola outbreak, the 2025 Annual Report, clinical trials in the European Union, preparations for new legislation, and an update from the Network Data Steering Group.

And the agency’s Committee for Medicinal Products for Human Use released its meeting summary on June 26, announcing recommendations for 6 new medicines.

The committee also announced it has revoked marketing authorization for Tavneos (avacopan), which is used to treat the rare inflammatory conditions, granulomatosis with polyangiitis or microscopic polyangiitis, because its benefits outweigh its risks.

In the United Kingdom, the UK Medicines and Healthcare products Regulatory Agency (MHRA) announced a collaboration with the US FDAto establish dedicated, reciprocal liaison officer roles within each organization that will support scientific exchange and tackle regulatory challenges.

On June 11, MHRA approved the UK’s first GLP-1 tablet for weight loss and weight management.

And on June 12, MHRAapproved a new lower dose epinephrine nasal spray for the emergency treatment of anaphylaxis in children 4 years of age and older.

And finally, an MHRA report detailed the results of public consultation on the use of AI in healthcare, stating “There is a broad consensus that existing regulatory approaches need to be adapted to meet the rapid pace of change and development of AI technology.”

“It is an excellent effort from the MHRA in gathering all the input on the use of AI in healthcare from various sectors (patients, researchers, regulators, etc.) that will support the future creation of regulation and standard to ensure the safety and efficacy of the AI in healthcare and that it performs as intended in both pre-market and post-market phases,” independent GMP consultant, Rory Budihandojo, told 

. “This report can be leveraged by other agencies and even better if there’s a concerted effort between inter agencies in creatin a universal regulations that transverse all other regulations (e.g., GMP).”

Updates from Standards Setting Organizations in June 2026

In the world of standard-setting organizations, the US Pharmacopeia (USP) published product-specific biologic standards for public comment on June 1, 2026

and released their annual drug shortages report on June 9.

 are lasting longer, up from 2 years in 2019 and 4.3 years in 2024 to 5 years in 2025. Product discontinuation is also at a high level, with a 60% increase from 2024 to 2025. However, the total number of shortages decreased by 23% in 2025.

Source: USP Annual Drug Shortages Report (reference 22). Image credit: Drew Kazdin 

Responding to the USP’s new biologics standards, 

, managing director and consultant with Albanese Consulting, LLC, told PharmTech, that “Stakeholders should be aware that emerging product specific monographs for several biologics that USP has published are not official and not enforceable by the FDA. The emerging standards are based on publicly available scientific information to encourage stakeholder feedback. The monograph can only move to official status when published via the Pharmacopoeial Forum process for stakeholder comments and approved by the expert committee.” 

In Europe, EDQM released its annual report on public health in Europe

 and published guidelines on supply chain reporting.

For detailed coverage and expert insights regarding these stories, as well as a list of published guidance documents and upcoming regulatory events, visit PharmTech.com.

FDA accepts first in silico drug development tool under ISTAND program to help predict drug-induced liver injury. FDA. June 3, 2026. Accessed June 26, 2026. 

https://www.fda.gov/drugs/drug-alerts-and-statements/fda-accepts-first-silico-drug-development-tool-under-istand-program-help-predict-drug-induced-liver

Ensuring quality and access: FDA’s approach to generic drug oversight. FDA. June 8, 2026. Accessed June 26, 2026. 

https://www.fda.gov/news-events/fda-voices/ensuring-quality-and-access-fdas-approach-generic-drug-oversight

FDA broadens access to over-the-counter naloxone nasal spray for opioid overdose. FDA. June 16, 2026. Accessed June 26, 2026. 

https://www.fda.gov/news-events/press-announcements/fda-broadens-access-over-counter-naloxone-nasal-spray-opioid-overdose

FDA Approves New Indication for Tzield (teplizumab) for Certain Pediatric Patients with Recently Diagnosed Stage 3 Type 1 Diabetes. Press Release. FDA. June 15, 2026. Accessed June 29, 2026. 

https://www.fda.gov/news-events/press-announcements/fda-approves-new-indication-tzield-teplizumab-certain-pediatric-patients-recently-diagnosed-stage-3

FDA Approves Drug for Pediatric Stage 3 Type I Diabetes. FDA. June 12, 2026. Accessed June 29, 2026. 

https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-drug-pediatric-stage-3-type-i-diabetes

FDA approves belzutifan with pembrolizumab for adjuvant treatment of renal cell carcinoma. FDA. June 12, 2026. Accessed June 29, 2026. 

https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-belzutifan-pembrolizumab-adjuvant-treatment-renal-cell-carcinoma

FDA approves capivasertib with abiraterone and prednisone for PTEN-deficient androgen pathway modulation-naïve or -sensitive prostate cancer. Press release. FDA. June 12, 2026. 

https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-capivasertib-abiraterone-and-prednisone-pten-deficient-androgen-pathway-modulation

FDA approves palbociclib with trastuzumab, with or without pertuzumab, and endocrine therapy for the maintenance treatment of HR-positive, HER2-positive metastatic breast cancer. Press release. FDA. June 24, 2026. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-palbociclib-trastuzumab-or-without-pertuzumab-and-endocrine-therapy-maintenance

FDA Actions to Accelerate and Modernize Early and Late-Stage Clinical Development. FDA. June 22, 2026. 

https://www.fda.gov/industry/fda-actions-accelerate-and-modernize-early-and-late-stage-clinical-development

Lessons Learned from our Roundtable with Rare Disease Advocates. FDA Voices blog post. FDA. June 18, 2206. https://www.fda.gov/news-events/fda-voices/lessons-learned-our-roundtable-rare-disease-advocates

EMA, AMA and African regulatory authorities join forces on Ebola outbreak response. Press release. EMA. June 3, 2026. 

https://www.ema.europa.eu/en/news/ema-ama-african-regulatory-authorities-join-forces-ebola-outbreak-response

EMA’s 2025 annual report shows strong approval numbers for human and veterinary medicines. Press release. EMA. June 11, 2026. 

https://www.ema.europa.eu/en/news/emas-2025-annual-report-shows-strong-approval-numbers-human-veterinary-medicines

Meeting highlights from the Pharmacovigilance Risk Assessment Committee (PRAC) 8-11 June 2026. Press release. EMA. June 12, 2026. 

https://www.ema.europa.eu/en/news/meeting-highlights-pharmacovigilance-risk-assessment-committee-prac-8-11-june-2026

EMA Management Board: highlights of June 2026 meeting. Press release. EMA. June 15, 2026. 

https://www.ema.europa.eu/en/news/ema-management-board-highlights-june-2026-meeting

Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 22-25 June 2026. Press release. EMA. June 26, 2026. https://www.ema.europa.eu/en/news/meeting-highlights-committee-medicinal-products-human-use-chmp-22-25-june-2026

EMA recommends revoking marketing authorisation for Tavneos. Press release. EMA. June 26, 2026. https://www.ema.europa.eu/en/news/ema-recommends-revoking-marketing-authorisation-tavneos

Unique liaison programme set to reinforce close collaboration between MHRA and FDA. Press release. MHRA. June 15, 2026. 

https://www.gov.uk/government/news/unique-liaison-programme-set-to-reinforce-close-collaboration-between-mhra-and-fda

First GLP-1 tablet for weight loss approved in the UK. MHRA. Press release. June 11, 2026. 

https://www.gov.uk/government/news/first-glp-1-tablet-for-weight-loss-approved-in-the-uk

Lower dose needle-free allergy treatment approved for younger children. Press release. MHRA. June 12, 2026. 

https://www.gov.uk/government/news/lower-dose-needle-free-allergy-treatment-approved-for-younger-children

MHRA landmark report reveals public views on AI in healthcare. Press release. MHRA. June 11, 2026.https://www.gov.uk/government/news/mhra-landmark-report-reveals-public-views-on-ai-in-healthcare

USP Publishes Product-Specific Emerging Biologics Standards for Public Comment. Press Release. USP. June 1, 2026. 

https://www.usp.org/news/usp-publishes-product-specific-emerging-biologics-standards-for-public-comment

USP Annual Drug Shortages Report finds rising discontinuations, hidden upstream risk. Press release. USP. June 9, 2026 https://www.usp.org/news/usp-2026-annual-drug-shortages-report-rising-discontinuations-supply-chain-risk

The EDQM highlights its impact on public health in Europe and beyond in its 2025 annual report. Press release. EDQM. June 22, 2026. 

https://www.edqm.eu/en/-/the-edqm-highlights-its-impact-on-public-health-in-europe-and-beyond-in-its-2025-annual-report

EDQM publishes guidelines to increase reporting on disappearances of medicinal products from the legal supply chain. Press release. EDQM. June 18, 2026. https://www.edqm.eu/en/-/edqm-publishes-guidelines-to-increase-reporting-on-disappearances-of-medicinal-products-from-the-legal-supply-chain

Regulatory Guidance Documents Published in June 2026

Drug and Device Manufacturer Communications With Payors, Formulary Committees, and Similar Entities – Questions and Answers

https://www.fda.gov/regulatory-information/search-fda-guidance-documents/drug-and-device-manufacturer-communications-payors-formulary-committees-and-similar-entities

Certain Postapproval Requirements and Resources for ANDAs 

https://www.fda.gov/regulatory-information/search-fda-guidance-documents/certain-postapproval-requirements-and-resources-andas

FDA Issues Draft Guidance to Help Accelerate Cell and Gene Therapies for Patients. 

https://www.fda.gov/news-events/press-announcements/fda-issues-draft-guidance-help-accelerate-cell-and-gene-therapies-patients

Forms FDA 3542a and FDA 3542: Questions and Answers


https://www.fda.gov/regulatory-information/search-fda-guidance-documents/forms-fda-3542a-and-fda-3542-questions-and-answers

Guidance, Master Protocols for Drug and Biological Product Development

https://www.fda.gov/regulatory-information/search-fda-guidance-documents/master-protocols-drug-and-biological-product-development

Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products, Draft Guidance for Industry

https://www.fda.gov/regulatory-information/search-fda-guidance-documents/demonstrating-substantial-evidence-effectiveness-human-drug-and-biological-products

Submitting Clinical Trial Datasets to Evaluate the Impact of Immunogenicity on the Pharmacokinetics of a Drug

https://www.fda.gov/regulatory-information/search-fda-guidance-documents/submitting-clinical-trial-datasets-evaluate-impact-immunogenicity-pharmacokinetics-drug

Quantitative Systems Pharmacology (QSP)-Based Dose Selection for Minimum Anticipated Biological Effect Level (MABEL) in First-in-Human (FIH) Trials


https://www.fda.gov/regulatory-information/search-fda-guidance-documents/quantitative-systems-pharmacology-qsp-based-dose-selection-minimum-anticipated-biological-effect

Hubei Gedian Humanwell Pharmaceutical Co., Ltd 

https://www.fda.gov/inspections-compliance-enforcement-and-criminal-investigations/warning-letters/hubei-gedian-humanwell-pharmaceutical-co-ltd-725591-06032026

https://www.fda.gov/inspections-compliance-enforcement-and-criminal-investigations/warning-letters/fagron-bv-724551-05122026

ClinicalTrials.gov: Essentials for Academic Medical Centers


https://www.fda.gov/drugs/news-events-human-drugs/clinicaltrialsgov-essentials-academic-medical-centers-07142026

July 23-24, 2026: Meeting of the Pharmacy Compounding Advisory Committee


https://www.fda.gov/advisory-committees/advisory-committee-calendar/july-23-24-2026-meeting-pharmacy-compounding-advisory-committee-07232026

FDA/Health and Environmental Sciences Institute/Safety Pharmacology Society Hybrid Workshop: ICH S7A and New Approach Methodologies (NAMs) in Safety Pharmacology


https://www.fda.gov/drugs/news-events-human-drugs/fdahealth-and-environmental-sciences-institutesafety-pharmacology-society-hybrid-workshop-ich-s7a

Accelerating Product Development for Pediatric Systemic Lupus Erythematosus (SLE)


https://www.fda.gov/drugs/news-events-human-drugs/accelerating-product-development-pediatric-systemic-lupus-erythematosus-sle-07302026

EMA multi-stakeholder workshop on supporting innovation in cardiovascular medicines and medical devices in the EU


https://www.ema.europa.eu/en/events/ema-multi-stakeholder-workshop-supporting-innovation-cardiovascular-medicines-medical-devices-eu

Sixteenth industry stakeholder platform on research and development support


https://www.ema.europa.eu/en/events/sixteenth-industry-stakeholder-platform-research-development-support

Managing product data quality in PMS: processes, known issues, current status and best practices


https://www.ema.europa.eu/en/events/managing-product-data-quality-pms-processes-known-issues-current-status-best-practices

What to Know About Regulations in June 2026

Blood drop, floating stem cells. Hematopoietic stem cell collection, regenerative medicine, blood-derived stem cells, donor therapy, transplantation concept | Image Credit: ©Елена Бутусова -stock.adobe.com

The FDA announced on June 30, 2026 that it has approved Tregzi for the treatment of chronic graft-versus-host disease (GVHD), a condition in which transplanted donor blood cells attack the recipient's body.

 Tregzi, a donor-derived cellular immunotherapy developed by Orca Biosystems, is the first regulatory T-cell-based immunotherapy approved for improving chronic GVHD-free survival in this patient population, marking a shift in how transplant centers might approach an outcome that has historically been difficult to prevent through pharmacologic means alone.

"For patients with blood cancers who need stem cell transplantation, chronic graft-versus-host disease has long been one of the most feared and difficult-to-prevent complications," said Karim Mikhail, B.Pharm., M.S., acting director of the Center for Biologics Evaluation and Research, in a press release.

 "Today's approval offers a genuine new approach that can help reconstitute the immune system while substantially reducing that risk and reflects the promise of what cellular therapy can deliver for patients."

“For transplant physicians, one of our greatest challenges has long been preserving the vital graft-versus-leukemia effect while minimizing the risk of GVHD and infection,” said Miguel-Angel Perales, M.D., medical oncologist and chief of the Adult Bone Marrow Transplant Service at Memorial Sloan Kettering Cancer Center, in a press release.

 “The FDA approval of TREGZI signals a new era in transplant medicine. This precision-engineered cell therapy is built on the foundational principles established by our early CD34 cell selection work and can now be delivered at scale, equipping providers with a new option to reduce serious toxicities and improve treatment outcomes."

How the Cell Therapy Is Manufactured and Administered

Unlike a standard stem cell transplant, which typically infuses a single population of donor cells, Tregzi is composed of 3 distinct cell components manufactured from a single donor collection: purified hematopoietic stem and progenitor cells, regulatory T cells, and conventional T cells. All 3 are derived from the mobilized peripheral blood of a donor who is an 8/8 human leukocyte antigen match, either a relative or an unrelated donor.

Regulatory T cells are immune cells that help regulate immune responses and maintain immune tolerance. By isolating and formulating this cell type alongside the stem and progenitor cells and conventional T cells, the therapy is intended to support reconstitution of a patient's blood-forming and immune systems while lowering the likelihood that donor-derived cells will attack healthy tissue. Patients receive the treatment after a course of chemotherapy that prepares the body for the transplant.

"We founded Orca Bio on the audacious goal to engineer living cells into curative medicines, rooted in the belief that single-cell precision could fundamentally rewrite patient outcomes," said Nate Fernhoff, PhD, co-founder and chief executive officer of Orca Bio, in a press release.

 "The FDA approval of TREGZI is a significant milestone that stands on the shoulders of decades of pioneering science. As we enter this next chapter, our focus turns to the immense responsibility of delivering TREGZI reliably, precisely and safely to the patients and families counting on us."

Trial Data Behind the Chronic GVHD-Free Survival Endpoint

The approval was based on findings from PRECISION-T, a randomized controlled trial that enrolled 187 adult patients with blood cancers, including acute leukemia and myelodysplastic syndrome. Participants were assigned to receive either Tregzi or a standard stem cell transplant, and the trial's primary endpoint was chronic GVHD-free survival. This was measured as the time from transplantation to the earliest occurrence of either death from any cause or the first onset of moderate or severe chronic GVHD, tracked within 2 years of the transplant date.

After 1 year, 78% of patients who received Tregzi met this endpoint, compared with 38.4% of those who received a standard transplant. When death was accounted for as a competing risk, 12.6% of Tregzi recipients developed serious chronic GVHD within a year, versus 44% among patients who received a standard transplant. According to FDA, the trial's results demonstrate clinical benefit for the studied population; therefore, the agency concluded that Tregzi's benefits outweigh its risks.

The safety profile observed in the trial was consistent with patients undergoing stem cell transplantation, with infection as the most common adverse event. No  severe reactions occurred during infusion, and no cases of graft failure were reported during the study period. 

“Developing this concept from early foundational research in our labs based upon the fundamental biology of regulatory T cells, to it now receiving the first FDA approval for a therapy that utilizes highly purified Tregs, is a defining moment for the transplant community," said Robert Negrin, M.D., professor of medicine, blood and marrow transplantation at Stanford Medicine, in a press release.

 "The peer-reviewed findings demonstrated this precision-engineered cell therapy delivered improved GVHD-free survival alongside less toxicity, including fewer serious infections and lower non-relapse mortality."

The application received both Orphan Drug and Regenerative Medicine Advanced Therapy designations ahead of its approval.

FDA approves new treatment that uses donor immune cells to prevent serious complications in blood cancer patients. Press release. FDA. June 30, 2026. 

https://www.fda.gov/news-events/press-announcements/fda-approves-new-treatment-uses-donor-immune-cells-prevent-serious-complications-blood-cancer

Orca Bio’s TREGZI™ receives U.S. FDA approval as first and only precision-engineered cell therapy for allogeneic transplant in adults with hematological malignancies. Press release. Orca Bio. June 30, 2026. 

https://orcabio.com/orca-bios-tregzi-receives-u-s-fda-approval-as-first-and-only-precision-engineered-cell-therapy-for-allogeneic-transplant-in-adults-with-hematological-malignanciesorca-bio-adds-east/

Articles

FDA approval of Tregzi introduces the first regulatory T-cell–based donor-derived cellular immunotherapy to improve chronic GVHD-free survival after allogeneic transplant in adults with hematologic malignancies. The product is manufactured from a single 8/8 HLA-matched donor mobilized peripheral blood collection into three components—purified HSPCs, regulatory T cells, and conventional T cells—administered following conditioning chemotherapy. In the randomized PRECISION-T trial, Tregzi markedly reduced moderate-to-severe chronic GVHD and improved the composite survival endpoint, with a transplant-consistent safety profile.

FDA has approved Tregzi, the first regulatory T-cell therapy shown to reduce chronic GVHD and improve survival in blood cancer transplant patients.

FDA Grants First-in-Class Approval for Treg Cell Therapy in Blood Cancer Transplant Patients

Macro closeup of young woman face portrait in profile side with Grave's disease hyperthyroidism symptoms of ophthalmopathy bulging eyes proptosis edema | Image Source: ©Andriy Blokhin -stock.adobe.com

Roche announced on June 30, 2026 that FDA granted priority review to the company’s supplemental biologics license application (sBLA) for satralizumab (Enspryng) for the treatment of thyroid eye disease (TED).

The agency accepted the filing after 2 randomized, placebo-controlled global phase 3 SatraGO studies found consistent improvements of TED symptoms and a more favorable safety profile when compared with currently available treatments. The study data were presented at the American Society of Ophthalmic Plastic and Reconstructive Surgery in October 2025.

“The FDA’s decision to grant priority review to Enspryng is an important step toward expanding treatment options for people living with thyroid eye disease,” said Levi Garraway, MD, PhD, Roche’s chief medical officer and head of Global Product Development, in a press release.

 “By targeting the underlying disease biology with a novel mechanism of action, this subcutaneous therapy has the potential to introduce a new treatment approach that combines clinical efficacy and a favorable safety profile with the convenience of at-home administration.”

TED is progressive, rare inflammatory autoimmune disease that can threaten sight. Symptoms include redness, swelling, eyelid retraction, eye bulging, double vision, and pain. TED impacts approximately 155 out of every 100,000 people and commonly occurs in people with hyperthyroidism.

Satralizumab is a humanized monoclonal antibody that targets IL-6, which is a messenger in the body’s inflammatory response, receptor activity, according to Roche. It was created using novel recycling antibody technology that allows for sustained IL-6 inhibition by binding to the IL-6 receptor.

Satralizumab is currently approved for the treatment of neuromyelitis optica spectrum disorder in 90 countries, including the United States and the European Union, where it has orphan drug designation. Roche is developing the treatment for additional neurological autoimmune and inflammatory diseases, including autoimmune encephalitis and myelin oligodendrocyte glycoprotein antibody-associated disease.

Roche Launches AXELIOS 1 Sequencing Platform

In other company news, on June 29, 2026, Roche launched its next-generation single-molecule sequencing platform, AXELIOS 1, powered by sequencing by expansion (SBX) technology. “The platform converts DNA/RNA into high signal‑to‑noise 'Xpandomers,' which are measured by a sequencing instrument using a reusable complementary metal–oxide–semiconductor sensor containing millions of nanopores. This architecture enables accurate, ultra‑rapid, flexible and high‑throughput, single‑molecule sequencing with near real‑time analysis,” the company stated in a press release.

“AXELIOS 1 will deliver a disruptive sequencing solution that combines high accuracy with unprecedented speed and scalability. These attributes, combined with our high level of cost efficiency, will enable the sequencing community to develop applications that previously were not feasible,” said Matt Sause, CEO of Roche Diagnostics, in a press release.

 “Additionally, in the future, AXELIOS 1 has the same potential to enable the next generation of clinical applications and unlock new frontiers in personalized healthcare.”

The new platform can be used for smaller, faster batches or larger projects and can produce whole-genome results in the same day. The flexible open setup gives researchers freedom to explore and create new workflows to gain knowledge of complex diseases.

Library prep kits and free open-source bioinformatics analysis suite will be offered by Roche. The platform can also be built for use of other life sciences tools.

FDA grants Priority Review to Roche’s Enspryng, the first and only at-home subcutaneous treatment option for thyroid eye disease (TED). Press release. Roche. June 30, 2026. 

https://www.roche.com/media/releases/med-cor-2026-06-30

Briceño C, et al. Primary Results of the Phase 3 SatraGO-1 and SatraGO-2 Trials: Efficacy and Safety of Satralizumab in Thyroid Eye Disease. Presented at the American Society of Ophthalmic Plastic and Reconstructive Surgery (ASOPRS) Fall Scientific Symposium, October 16–17, 2025.

Roche announces the launch of AXELIOS 1, a transformative next-generation sequencing platform. Press release. Roche. June 29, 2026. 

https://www.roche.com/media/releases/med-cor-2026-06-29

FDA granted priority review to Roche’s sBLA for satralizumab (Enspryng) in thyroid eye disease, supported by two global randomized phase 3 SatraGO trials showing consistent symptom improvement and a comparatively favorable safety profile. The subcutaneous, potentially at-home regimen targets IL‑6 receptor signaling via recycling antibody technology to sustain inhibition. Satralizumab is already approved for neuromyelitis optica spectrum disorder in ~90 countries. Roche also launched AXELIOS 1, a single-molecule sequencing system using sequencing-by-expansion for rapid same-day whole genomes, with an FDA action date expected within months.

The company’s thyroid eye disease treatment will be the first at-home subcutaneous disease-modifying treatment if approved.

Roche Gets Priority Review for Enspryng; Announces Sequencing Platform

Ask the Expert: Regulatory Developments Impacting Biologics

, Siegfried Schmitt, vice president, Technical, at Parexel, and Susan Schniepp, distinguished fellow at Nelson Labs, discuss regulatory and compendial announcements affecting biologics manufacturers.

The conversation was prompted by a question a PharmTech reader seeking to understand the significance of new publications from the FDA, the US Pharmacopeia (USP), and European regulators. Schniepp notes that these regulatory announcements are arriving in close succession, which is notable and appears to reflect a coordinated effort to support the rapidly evolving biologics field.

 have been released without accompanying specifications.

Schniepp views this as a deliberate move to assist manufacturers by providing validated methods while leaving specification-setting to individual companies based on their specific product. This indicates USP is helping streamline chemistry, manufacturing, and controls (CMC) sections in regulatory filings without imposing a one-size-fits-all standard.

, which was agreed upon in 2025, focuses on reducing drug shortages and eliminating supply chain bottlenecks.

 Schniepp highlights the concern that some critical raw materials or components used in biologics may be available from only a single supplier. If that supplier experiences a disruption, it can affect the entire industry. The act aims to address these single-source vulnerabilities and strengthen supply chain resilience.

issued a draft guidance on leveraging prior knowledge

 in the development of human gene therapy products incorporating genome editing.

 Rather than requiring each company to build a completely independent platform from scratch, the guidance would allow manufacturers to reference previously published studies and data to support their CMC filings, which could be a significant potential time-saver for an area where knowledge is often concentrated among a small number of firms. Both speakers emphasize that this guidance is still in draft form, with a public comment deadline of September 1, 2026, and encourage manufacturers to submit their perspectives.

How Are Regulators Supporting Biologics Development?

Schmitt broadens the conversation to the underlying intent behind these developments. Biologics, he notes, are frequently life-saving treatments that regulators are motivated to help bring to market. The challenge is not just regulatory approval, but ensuring these therapies can actually reach patients. That involves rethinking supply chains and manufacturing models. For cell and gene therapies, in particular, traditional centralized manufacturing and long-distance shipping are often not viable options. Both experts point to distributed, decentralized manufacturing (i.e., producing therapies physically closer to patients) as a likely direction for the industry.

Schniepp states that, in some cases, the final steps of manufacturing may need to occur in a clinical or hospital setting, such as combining a lyophilized product with water for injection. This requires close coordination between manufacturers and healthcare providers and must be factored into regulatory planning.

Rather than viewing regulators as lagging behind innovation or creating unnecessary burdens, the FDA, EMA, and USP are trying to keep pace with a fast-moving field and actively supporting industry progress, according to Schmitt and Schniepp, which is a positive development for patients, companies, and the broader ecosystem alike.

Biologics manufacturers should pay close attention to these announcements and engage with the process by submitting comments to both the FDA on its draft guidance and to USP on its proposed methodologies. Regulatory input from industry practitioners helps shape practical, workable standards.

Click on the video above to watch the experts answer the question:

“My company manufactures biologics, and we saw some recent actions by FDA, USP, and the EU, how do these regulatory actions impact us as a biologics manufacturer?”

USP publishes product-specific emerging biologics standards for public comment. USP. Press release. June 1, 2026. 

EMA. EMA welcomes political agreement on the Critical Medicines Act

. News release. ec.europa.eu. 12 May 2026. https://ec.europa.eu/commission/presscorner/detail/en/ip_26_1017

https://health.ec.europa.eu/publications/proposal-critical-medicines-act_en

FDA issues draft guidance to help accelerate cell and gene therapies for patients. FDA. Press release. June 2, 2026. 

Susan J. Schniepp is distinguished fellow at Nelson Labs and a member of 

Siegfried Schmitt is vice president, Technical at Parexel and a member of 

This episode of Ask the Expert takes a look at recent regulatory actions impacting biologics manufacturers.

Regulatory Developments Impacting Biologics

Antibody drug conjugate in blue with four drug compounds linked to IgG immunoglobulin; ADC in blue against blue background 3d render | Image Credit:  ©huenstructurebio.com -stock.adobe.com

Lonza is expanding payload-linker and HPAPI manufacturing at its Visp, Switzerland site by upgrading its drug-linker center of excellence and adding commercial-scale capabilities within an existing GMP facility. The buildout integrates payload-linker production and purification into dedicated analytical and process development labs, enabling multipurpose, scale-out manufacturing with room for additional suites. The site already offers end-to-end ADC services spanning mAb manufacturing, conjugation, drug product, and QC for highly potent materials. Operations are targeted for 2028, supporting clinical-to-commercial supply.

The outsourcing company is adding commercial-scale capabilities to manufacture HPAPI and ADC payload-linkers.