Roflumilast

Barely a month after joining Merck & Co. through a $10-billion merger, Verona Pharma has withdrawn its EU marketing application for Ohtuvayre (ensifentrine), the inhaled PDE3/4 inhibitor touted as a key addition to Merck's cardio-pulmonary portfolio. The filing was pulled on Oct. 30 as the European Medicines Agency (EMA) was still evaluating initial information from the company, leaving the review process unfinished and without any recommendations from the regulator. Ohtuvayre was approved in the US last year for maintenance treatment of chronic obstructive pulmonary disease (COPD), supported by two Phase III trials demonstrating significant lung function improvements in patients with moderate-to-severe illness. Review of priorities However, the studies did not enroll COPD patients exacerbating on triple therapy, a group where some pulmonologists think it is likely to be used most often. In a recent FirstWord interview, one key opinion leader (KOL) described Ohtuvayre as effective and meaningfully safer than roflumilast, but suggested that this data gap in triple-therapy patients was likely to impact how often it will be used. For more, see – KOL Views Q&A: Leading expert impressed by Ohtuvayre but sees one feature weighing on use in COPD. According to the EMA, Verona stated that it withdrew the Ohtuvayre application "following a review of its priorities." Positive recommendations While Ohtuvayre's EU prospects appear to be on pause, the EMA's Committee for Medicinal Products for Human Use (CHMP) issued a number of positive opinions for other therapies on Friday. Nearly three months after getting the all-clear in the US, Ionis and Otsuka's Dawnzera (donidalorsen) received a CHMP recommendation for routine prevention of recurrent hereditary angioedema attacks in adults and adolescents aged 12 and older. The RNA-targeting prophylactic, administered subcutaneously once a month, is backed by Phase III data from the OASIS-HAE study showing it led to a mean 81% reduction in HAE attacks versus placebo. Otsuka holds exclusive rights to market Dawnzera across Europe and the Asia-Pacific region. CHMP also issued a positive opinion for Eli Lilly's Inluriyo (imlunestrant), an oral selective oestrogen receptor degrader (SERD) aimed at adult patients with locally advanced or metastatic breast cancer and an activating ESR1-mutation. The drug was approved by US regulators in September based on data from the Phase III EMBER-3 trial showing it lowered the risk of progression or death by 38% over standard endocrine therapy in ESR1-mutant patients. The risk-reduction rate ticked up to 43% when Inluriyo was given alongside Lilly's CDK4/6 inhibitor Verzenio (abemaciclib), compared to SERD alone (see – KOL Views Q&A: Battle for oral SERD supremacy in HR+ breast cancer boils down to efficacy). Sanofi's Teizeild (teplizumab) secured a favourable recommendation as a therapy intended to preserve beta-cell function and delay progression to stage 3 type 1 diabetes (T1D) in adults and children 8 years and older with stage 2 disease. In clinical testing, median time to onset of stage 3 T1D was 49.5 months with Teizeild versus 24.9 months for placebo. Sanofi gained the drug — marketed in the US as Tzield and approved there three years ago — through its nearly $3 billion takeout of Provention Bio in 2023. Meanwhile, Waskyra (etuvetidigene autotemcel) became the first gene therapy to receive a positive CHMP opinion for Wiskott-Aldrich syndrome, a rare immunodeficiency that almost exclusively affects males and can lead to bleeding problems and eczema as well as susceptibility to infections. Data from a clinical development programme involving 27 patients showed that Waskyra reduced both severe infections (from 2.0 to 0.12 annual events by years 2-3) and moderate-to-severe bleeding episodes (from 2.0 to 0.16 annual events by years 2-3) compared to pre-treatment rates. If authorised, Waskyra would offer an alternative for patients who are candidates for stem-cell transplantation, but lack suitable donors. The applicant is Fondazione Telethon ETS, which also submitted a filing to the FDA earlier this year. Anavex craters The news was less upbeat for Anavex Life Sciences, however, after EU regulators delivered a negative trend vote on its marketing application for blarcamesine in early Alzheimer's disease, signaling that the agency is likely to issue an unfavourable opinion when it meets in December. According to Anavex, the orally available drug candidate is designed to restore cellular homeostasis by targeting SIGMAR1 and muscarinic receptors. The regulatory setback triggered a sharp investor reaction, with Anavex shares falling roughly 38% on Friday. The company says it plans to seek a formal re-examination once the opinion is formally adopted, a process that would shift review to a separate CHMP panel and allow Anavex to submit additional biomarker analyses. The EU filing included data from the Phase IIb/III ANAVEX2-73-AD-004 trial showing that once-daily oral blarcamesine slowed clinical progression by 36.3% at 48 weeks — and by nearly 50% in patients with the prespecified SIGMAR1 wild-type genotype — on the primary ADAS-Cog13 cognitive measure. Separately, Anavex said Friday that the FDA has advised the company to schedule a meeting to discuss clinical results from its Alzheimer's trials.

Investigational ZORYVE cream 0.05% was formulated for the delicate skin of infants and young children with mild to moderate atopic dermatitis Topline results anticipated in Q1 2026 The U.S. FDA approved ZORYVE cream 0.05% for the treatment of mild to moderate atopic dermatitis in children ages 2 to 5 years in October 2025 Atopic dermatitis impacts 9.6 million children in the U.S., with up to 60% developing symptoms within their first year WESTLAKE VILLAGE, Calif., Nov. 13, 2025 (GLOBE NEWSWIRE) -- Arcutis Biotherapeutics, Inc. (Nasdaq: ARQT), a commercial-stage biopharmaceutical company focused on developing meaningful innovations in immuno-dermatology, today announced the enrollment of the last subject in the INTEGUMENT-INFANT Phase 2 open-label clinical study, which is evaluating the safety and tolerability of investigational once-daily ZORYVE (roflumilast) cream 0.05% in infants aged 3 months to less than 24 months with mild to moderate atopic dermatitis. ZORYVE cream is a highly selective and potent topical phosphodiesterase-4 (PDE4) inhibitor formulated to address the unique needs of individuals with atopic dermatitis. “Atopic dermatitis often presents in infancy and can cause significant discomfort from the itch and rash, and even disruption to sleep and social activities, which can negatively impact the affected child and the entire family,” said Patrick Burnett, MD, PhD, FAAD, chief medical officer at Arcutis. “Enrollment in the INTEGUMENT-INFANT study has been brisk, reflecting strong interest from parents seeking an alternative to topical corticosteroids. This clinical milestone represents a significant step in advancing a well-tolerated topical treatment for this vulnerable population, for whom FDA-approved options remain limited. We eagerly anticipate the study results as we continue to develop ZORYVE cream 0.05% as a potential new treatment for pediatric atopic dermatitis in infants as young as three months.” About the INTEGUMENT-INFANT StudyThe INTEGUMENT-INFANT Phase 2, open-label, multicenter study is evaluating the safety and tolerability of ZORYVE cream 0.05% applied once daily over a four-week period in 101 infants aged 3 months to less than 24 months with mild to moderate atopic dermatitis. The study builds upon the earlier Maximal Usage (MUSE) pharmacokinetics trial ARQ-151-105, which also evaluated ZORYVE cream 0.05% in this age group. Topline results from the study are expected to be reported in Q1 2026. About Atopic DermatitisAtopic dermatitis is the most common type of eczema, affecting approximately 9.6 million children and 16.5 million adults in the United States. Atopic dermatitis is a chronic, relapsing, and genetically predisposed inflammatory skin disease that has unique clinical presentations across the lifespan. The disease typically appears as a red, intensely itchy rash that can occur anywhere on the body. It presents differently in infants, children, and adults. Younger children typically have more widespread atopic dermatitis, including on the face, neck, and areas around the knees and elbows. Atopic dermatitis is often initially diagnosed during childhood with approximately 60% of children developing symptoms within their first year. Pediatric atopic dermatitis can negatively impact the entire family by significantly disrupting sleep, increasing the risk of skin infections, and leading to developmental and emotional strain for both the child and caregivers. About ZORYVE® (roflumilast) ZORYVE is the number one prescribed branded topical therapy across three major inflammatory dermatoses combined—atopic dermatitis, seborrheic dermatitis, and plaque psoriasis. ZORYVE is a topical formulation of roflumilast, an advanced targeted topical phosphodiesterase type 4 (PDE4) inhibitor. Inhibiting PDE4, an intracellular enzyme that is an established target in dermatology, decreases the production of pro-inflammatory mediators. This decreases inflammation in the skin and balances the skin’s immune system. Demonstrating both clinical impact and broad industry recognition, ZORYVE has been honored with multiple prestigious awards and recommendations. ZORYVE was recently awarded by Allure with the "2025 Best of Beauty Breakthrough Award," making it the first FDA-approved medication for atopic dermatitis, plaque psoriasis, and seborrheic dermatitis to win this prominent award. ZORYVE cream 0.3% and ZORYVE foam 0.3% were also awarded the National Psoriasis Foundation’s Seal of Recognition — the first FDA-approved prescription brand to receive the honor. Additionally, the American Academy of Dermatology (AAD) issued a strong recommendation for the use of ZORYVE cream 0.15% in adult patients with mild to moderate atopic dermatitis, according to updated guidelines released in June 2025. In 2024, ZORYVE cream 0.15% was awarded Glamour’s Beauty and Wellness Award for “Best Eczema Product.” About ArcutisArcutis Biotherapeutics, Inc. (Nasdaq: ARQT) is a commercial-stage medical dermatology company that champions meaningful innovation to address the urgent needs of individuals living with immune-mediated dermatological diseases and conditions. With a commitment to solving the most persistent patient challenges in dermatology, Arcutis has a growing portfolio of advanced targeted topicals approved to treat three major inflammatory skin diseases. Arcutis’ unique dermatology development platform coupled with our dermatology expertise allows us to develop differentiated therapies against biologically validated targets, and has produced a robust pipeline for a range of inflammatory dermatological conditions. For more information, visit www.arcutis.com or follow Arcutis on LinkedIn, Facebook, Instagram and X. INDICATIONSZORYVE cream, 0.05%, is indicated for topical treatment of mild to moderate atopic dermatitis in pediatric patients 2 to 5 years of age. ZORYVE cream, 0.15%, is indicated for topical treatment of mild to moderate atopic dermatitis in adult and pediatric patients 6 years of age and older. IMPORTANT SAFETY INFORMATION ZORYVE is contraindicated in patients with moderate to severe liver impairment (Child-Pugh B or C). The most common adverse reactions reported (≥1%) for ZORYVE cream 0.05% for pediatric patients with atopic dermatitis 2 to 5 years of age were upper respiratory tract infection (4.1%), diarrhea (2.5%), vomiting (2.1%), rhinitis (1.6%), conjunctivitis (1.4%), and headache (1.1%). The most common adverse reactions reported (≥1%) for ZORYVE cream 0.15% for patients with atopic dermatitis 6 years of age or older were headache (2.9%), nausea (1.9%), application site pain (1.5%), diarrhea (1.5%), and vomiting (1.5%). Please see full Prescribing Information for ZORYVE cream. Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. For example, statements contained in this press release regarding matters that are not historical facts are forward-looking statements. These statements are based on the Company’s current beliefs and expectations. Such forward-looking statements include, but are not limited to, statements regarding the potential for ZORYVE cream 0.05% as a treatment for pediatric AD patients, including infants from 3 to less than 24 months, and timing of potential clinical results. These statements are subject to substantial known and unknown risks, uncertainties and other factors that may cause our actual results, levels of activity, performance, or achievements to be materially different from the information expressed or implied by these forward-looking statements. Risks and uncertainties that may cause our actual results to differ include risks inherent in our business, reimbursement and access to our products, the impact of competition and other important factors discussed in the “Risk Factors” section of our Form 10-K filed with the U.S. Securities and Exchange Commission (SEC) on February 25, 2025, as well as any subsequent filings with the SEC. Any forward-looking statements that the company makes in this press release are made pursuant to the Private Securities Litigation Reform Act of 1995, as amended, and speak only as of the date of this press release. Except as required by law, we undertake no obligation to revise or update information herein to reflect events or circumstances in the future, even if new information becomes available. ContactsMediaAmanda Sheldon, Head of Corporate Communicationsmedia@arcutis.com InvestorsBrian Schoelkopf, Head of Investor Relationsir@arcutis.com