U.S. Food and Drug Administration Accepts Bristol Myers Squibb's New Drug Application for Iberdomide in Patients with Relapsed or Refractory Multiple Myeloma

Bristol Myers Squibb (NYSE: BMY) today announced that the U.S. Food and Drug Administration (FDA) has accepted a New Drug Application (NDA) for iberdomide combined with standard treatment (daratumumab + dexamethasone - IberDd) in patients with relapsed or refractory multiple myeloma (RRMM). Iberdomide is part of an investigational, new class of medicines called cereblon E3 ligase modulator (CELMoD) agents. The FDA has granted a Prescription Drug User Fee Act (PDUFA) date of August 17, 2026 for this indication.

• Iberdomide has the potential to be the first approved CELMoD agent
• The U.S. FDA has granted Breakthrough Therapy Designation and Priority Review for this indication and assigned a target action date of August 17, 2026

“The FDA’s acceptance of this application is a testament to the potential of iberdomide, in combination with anti-CD38 monoclonal antibodies, as a novel, potent, oral treatment option, with a manageable safety profile, for patients with multiple myeloma,” said Cristian Massacesi, executive vice president and chief medical officer, Bristol Myers Squibb. “Furthermore, our filing for iberdomide based on the MRD endpoint, underscores our commitment to pioneering new ways of advancing life-saving therapies for patients living with cancer.”

The filing was based on results from a planned analysis of MRD negativity rates in the Phase 3 EXCALIBER-RRMM study evaluating iberdomide as a treatment for RRMM patients. The EXCALIBER-RRMM trial is ongoing and patients continue to be evaluated for progression-free survival (PFS).

The FDA also granted Breakthrough Therapy designation for iberdomide based on these data.

This review is being conducted under the FDA’s Project Orbis initiative, which enables concurrent review by the health authorities in several other countries.

https://en.wikipedia.org/wiki/Iberdomide

U.S. Food and Drug Administration Accepts Bristol Myers Squibb's New Drug Application for Iberdomide in Patients with Relapsed or Refractory Multiple Myeloma

FDA Approves Rhapsido (remibrutinib) for the Treatment of Chronic Spontaneous Urticaria

Novartis announced today that Rhapsido (remibrutinib) received US Food and Drug Administration (FDA) approval as an oral treatment for adult patients with chronic spontaneous urticaria (CSU) who remain symptomatic despite H1 antihistamine treatment. Rhapsido is a pill taken twice daily and does not require injections or lab monitoring. It is the first FDA-approved Bruton's tyrosine kinase inhibitor (BTKi) for CSU. Rhapsido helps to inhibit the release of histamine and other proinflammatory mediators by targeting BTK, offering a unique approach to CSU treatment.

"CSU is a serious disease that can cause debilitating symptoms and unpredictable flares. It's difficult to diagnose and manage," said Mark Lebwohl, MD, Dean for Clinical Therapeutics at the Icahn School of Medicine at Mount Sinai and member of the steering committee for the remibrutinib REMIX Phase III clinical trial program. "Remibrutinib represents a new way of treating CSU. By blocking the activity of BTK, remibrutinib stops a key pathway of the immune response in CSU. This is an exciting new option that has the potential to help a broad range of patients get fast relief."

CSU is a mast cell-driven condition thought to be caused by immune dysregulation. In people with CSU, the immune system can become activated through allergic (IgE) or autoimmune (IgG) pathways.4 This causes certain immune cells—mast cells and basophils—to activate the BTK protein. While not fully understood, it is believed that once activated, BTK leads to the release of histamine and other proinflammatory mediators that may cause the red, swollen, and itchy hives commonly seen in CSU.5,6

CSU symptoms are unpredictable, recurring for six weeks or more without an identified cause.7 Diagnosis can take up to 24 months.8 Many CSU patients say their symptoms negatively impact their sleep, work, and mental health.9,10,11 Antihistamines are the first-line treatment, but over half of patients still have symptoms, even at higher doses.2 Injectable treatments exist for those who don't respond to antihistamines, yet fewer than 20% of eligible patients receive them.12

"The approval of remibrutinib is an important development in CSU care. It quickly reduces symptoms, offering patients control of the hives and itching that they experience on a daily basis," said Giselle Mosnaim, MD, MS, an Allergist and Immunologist from Endeavor Health, Clinical Associate Professor at the University of Chicago Pritzker School of Medicine and REMIX trial investigator. "This is significant because it expands beyond existing injectable treatments and gives patients an oral option that can easily be incorporated into their daily lives."

"Many CSU patients feel misunderstood and settle for treatments that don't fully meet their needs," said Lynda Mitchell, CEO of Allergy & Asthma Network. "We support new treatment options that empower patients to choose what works best for them. This convenient new oral therapy offers a promising new way to manage CSU and potentially improve daily life for those living with this challenging condition."

Clinical data supporting approval

The FDA approval of Rhapsido in CSU is based on results from the Phase III REMIX-1 (NCT05030311) and REMIX-2 (NCT05032157) clinical trials in patients who remained symptomatic on second-generation H1 antihistamines. Rhapsido demonstrated superiority in change from baseline versus placebo in itch (ISS7), hives (HSS7), and weekly urticaria activity (UAS7) at Week 12.13 Significantly more patients treated with Rhapsido versus placebo achieved well-controlled disease (UAS7≤6) as early as Week 2 and at Week 12, and about one-third of patients achieved complete absence of itch and hives at Week 12.13 Rhapsido has a demonstrated safety profile that requires no lab monitoring.13 The most common adverse events (incidence ≥3%) were nasal congestion, sore throat, and runny nose (nasopharyngitis), bleeding, headache, nausea, and abdominal pain.13

Novartis has completed regulatory submissions for Rhapsido for the treatment of CSU across many countries, including in the European Union, Japan, and China, with priority review granted in China.

Transforming care in Immunology

"This approval of Rhapsido as the first and only BTK inhibitor in CSU is an important milestone in our journey to reshape care for overlooked immune-related conditions and offer more patients the potential to find fast relief," said Victor Bultó, President, US, Novartis. "Building on our legacy in advancing the treatment of allergic, dermatologic, and rheumatologic conditions, we are deeply committed to further investing in innovative, patient-focused therapies across immunology."

Discovered and developed by Novartis to target the BTK pathway as a driver of inflammation, remibrutinib is being investigated in ongoing clinical trials across a variety of immune-related conditions, including chronic inducible urticaria (CIndU), hidradenitis suppurativa (HS), and food allergy.

Rhapsido (remibrutinib) Indication

Rhapsido is a prescription medicine used to treat adults with chronic spontaneous urticaria (CSU) who continue to have symptoms that are not controlled with antihistamine treatment. Rhapsido should not be used to treat any other forms of hives (urticaria). It is not known if Rhapsido is safe and effective in children.

Rhapsido Important Safety Information

Before taking Rhapsido, patients should tell their health care provider about all of their medical conditions, including if they:

Have had a recent surgery or plan to have surgery. Health care providers may tell patients to stop taking Rhapsido for at least 3 to 7 days before and after any planned medical or surgical procedures

Have bleeding problems or are taking a blood thinner medicine

Have liver problems

Are pregnant or plan to become pregnant. It is not known if Rhapsido will harm an unborn baby. If patients become pregnant during treatment with Rhapsido, they should talk to their health care provider about registering in the pregnancy exposure registry for Rhapsido

Are breastfeeding or plan to breastfeed. It is not known if Rhapsido passes into breast milk

Have recently received or are scheduled to receive an immunization (vaccine). Patients should talk to their health care provider about receiving any immunizations before taking Rhapsido. They should not receive live or live-attenuated vaccines during treatment with Rhapsido

Patients should tell their health care provider about all the medicines they take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Taking Rhapsido with certain other medications may affect how Rhapsido or other medications work, and can cause side effects.

https://en.wikipedia.org/wiki/Remibrutinib

FDA Approves Rhapsido (remibrutinib) for the Treatment of Chronic Spontaneous Urticaria

FDA Approves Lasix ONYU (furosemide) for Treatment of Edema in Heart Failure

SQ Innovation, Inc. today announced that the U.S. Food and Drug Administration (FDA) has approved its drug-device combination Lasix ONYU (furosemide injection) for the treatment of edema (due to fluid overload) in adult patients with chronic heart failure. Lasix ONYU was developed to enable subcutaneous infusion of furosemide outside the healthcare setting for selected patients, as prescribed by a clinician without the need for a healthcare professional to administer the drug.

About 6.7 million Americans suffer from heart failure, with the prevalence expected to rise to 8.7 million by 2030. Heart failure is a leading cause of hospitalizations for individuals aged 65 and older with approximately 1.2 million hospitalizations per year [1].

Lasix ONYU consists of a novel high-concentration formulation of furosemide combined with a state-of-the-art small Infusor for treatment at home. The innovative design includes a reusable unit that can be used for 48 treatments and a plastic sterile single-use unit that is discarded after treatment. The two-component design reduces manufacturing complexity and cost, allowing the product to be offered at a different, more favorable price point which is expected to reduce barriers to widespread adoption.

“Lasix ONYU has the potential to be transformative in the care of patients experiencing worsening heart failure due to fluid overload,” said Pieter Muntendam, MD, founder, President and CEO of SQ Innovation. “Treating selected patients at home offers important benefits to patients, health systems and payors. We look forward to launching Lasix ONYU with leading health systems in the 4th quarter of 2025.”

Bioavailability and diuretic response were determined in a clinical study in which Lasix ONYU demonstrated complete bioavailability (112%) resulting in similar diuresis (115%) and natriuresis (117%) when compared to the same dose given by IV bolus. The biphasic delivery of furosemide by the Infusor resulted in a tempered diuretic response while IV bolus administration led to a shorter period of more intense diuresis. The results of the study were published in a leading cardiovascular journal [2].

“Heart failure is the most common serious medical condition in the U.S. and affects about one in four Americans during their lifetime. The number of patients affected is expected to double over the next 20 years and we currently already often lack adequate resources to take care of the 6.7 million patients affected presently – there are not enough beds, clinicians and funds”, said Dr. Javed Butler, Professor of Medicine at University of Mississippi and President, Baylor Scott and White Research Institute. “The only two actionable solutions now are more widespread adoption of guideline directed medical therapy (GDMT) and treating more patients at home with products such as subcutaneous diuretics instead of hospitalization for intravenous diuretics.”

“Decongestion through use of IV diuretics has been the cornerstone of treatment for reducing edema and hypervolemia in heart failure patients for over five decades,” stated S. Craig Thomas, Immediate Past President of the American Association of Heart Failure Nurses (AAHFN), an organization dedicated to advancing nursing education, clinical practice, and research to improve outcomes for heart failure patients. “The availability of accessible, affordable, and novel options that do not require the presence of a healthcare professional allows for transformative new clinical care-delivery. This means patients who now would typically need to be hospitalized for several days of IV treatment can instead remain home, supported by periodic or remote monitoring. The significance of this shift away from inpatient care for patients, hospitals, and payers cannot be overstated.”

Starting this quarter, Lasix ONYU will be available from leading pharmaceutical distributors enabling timely availability at participating medical facilities and affiliated retail pharmacies.

SQ Innovation is hosting a Conference Call and Webcast on Thursday October 9, at 4:30pm ET to introduce the product and answer questions from the community. Participating in the conference call will be:

Pieter Muntendam, MD, President and CEO of SQ Innovation

Mustafa M. Ahmed, MD, Professor of Medicine, Section Chief, Heart Failure, University of Florida Health, Gainesville, FL

Craig Thomas, Nurse Practitioner, Advanced Heart Failure Center, University of Virginia Health System, Charlottesville, VA and Immediate Past President American Association of Heart Failure Nurses (AAHFN)

https://en.wikipedia.org/wiki/Furosemide

FDA Approves Lasix ONYU (furosemide) for Treatment of Edema in Heart Failure

Aldeyra Therapeutics Announces FDA Acceptance for Review of Reproxalap New Drug Application for the Treatment of Dry Eye Disease

Aldeyra Therapeutics, Inc. (Nasdaq: ALDX) (Aldeyra)  announced   the U.S. Food and Drug Administration (FDA)   acceptance the  review of the resubmitted New Drug Application (NDA) for topical ocular reproxalap, a first-in-class investigational new drug candidate, for the treatment of the signs and symptoms of dry eye disease. The FDA assigned a Prescription Drug User Fee Act (PDUFA) target action date of December 16, 2025.

“Based on the FDA’s requirement for an additional clinical trial demonstrating the efficacy of reproxalap in treating the symptoms of dry eye disease, and per agreement with the FDA, the NDA resubmission contained a single clinical trial that achieved the primary endpoint of reducing ocular discomfort relative to the vehicle control,” stated Todd C. Brady, M.D., Ph.D., President and Chief Executive Officer of Aldeyra. “We look forward to a productive dialog with the FDA during the NDA review of reproxalap, which, to our knowledge, remains the only dry eye disease investigational therapy to have demonstrated acute activity in reducing ocular discomfort and redness in pivotal trials simulating the disease flares that are likely the most bothersome aspects of dry eye disease.”

About Reproxalap

Reproxalap is an investigational new drug candidate in development for the treatment of dry eye disease and allergic conjunctivitis, two of the largest markets in ophthalmology. Reproxalap is a first-in-class small-molecule modulator of RASP, which are elevated in ocular and systemic inflammatory diseases. The mechanism of action of reproxalap has been supported by the demonstration of statistically significant and clinically relevant activity in multiple physiologically distinct late-phase clinical indications. Reproxalap has been studied in more than 2,900 patients with no observed safety concerns; mild and transient instillation site irritation is the most commonly reported adverse event in clinical trials.

https://go.drugbank.com/drugs/DB16688

FDA Approves Zulresso (brexanolone) for the Treatment of Postpartum Depression

U.S. Food and Drug Administration   approved Zulresso (brexanolone) injection for intravenous (IV) use for the treatment of postpartum depression (PPD) in adult women. This is the first drug approved by the FDA specifically for PPD.

"Postpartum depression is a serious condition that, when severe, can be life-threatening. Women may experience thoughts about harming themselves or harming their child. Postpartum depression can also interfere with the maternal-infant bond. This approval marks the first time a drug has been specifically approved to treat postpartum depression, providing an important new treatment option," said Tiffany Farchione, M.D., acting director of the Division of Psychiatry Products in the FDA’s Center for Drug Evaluation and Research. "Because of concerns about serious risks, including excessive sedation or sudden loss of consciousness during administration, Zulresso has been approved with a Risk Evaluation and Mitigation Strategy (REMS) and is only available to patients through a restricted distribution program at certified health care facilities where the health care provider can carefully monitor the patient."

PPD is a major depressive episode that occurs following childbirth, although symptoms can start during pregnancy. As with other forms of depression, it is characterized by sadness and/or loss of interest in activities that one used to enjoy and a decreased ability to feel pleasure (anhedonia) and may present with symptoms such as cognitive impairment, feelings of worthlessness or guilt, or suicidal ideation.

Zulresso will be available only through a restricted program called the Zulresso REMS Program that requires the drug be administered by a health care provider in a certified health care facility. The REMS requires that patients be enrolled in the program prior to administration of the drug. Zulresso is administered as a continuous IV infusion over a total of 60 hours (2.5 days). Because of the risk of serious harm due to the sudden loss of consciousness, patients must be monitored for excessive sedation and sudden loss of consciousness and have continuous pulse oximetry monitoring (monitors oxygen levels in the blood). While receiving the infusion, patients must be accompanied during interactions with their child(ren). The need for these steps is addressed in a Boxed Warning in the drug’s prescribing information. Patients will be counseled on the risks of Zulresso treatment and instructed that they must be monitored for these effects at a health care facility for the entire 60 hours of infusion. Patients should not drive, operate machinery, or do other dangerous activities until feelings of sleepiness from the treatment have completely gone away.

The efficacy of Zulresso was shown in two clinical studies in participants who received a 60-hour continuous intravenous infusion of Zulresso or placebo and were then followed for four weeks. One study included patients with severe PPD and the other included patients with moderate PPD. The primary measure in the study was the mean change from baseline in depressive symptoms as measured by a depression rating scale. In both placebo controlled studies, Zulresso demonstrated superiority to placebo in improvement of depressive symptoms at the end of the first infusion. The improvement in depression was also observed at the end of the 30-day follow-up period.

The most common adverse reactions reported by patients treated with Zulresso in clinical trials include sleepiness, dry mouth, loss of consciousness and flushing. Health care providers should consider changing the therapeutic regimen, including discontinuing Zulresso in patients whose PPD becomes worse or who experience emergent suicidal thoughts and behaviors.

FDA Approves Zulresso (brexanolone) for the Treatment of Postpartum Depression

https://en.wikipedia.org/wiki/Allopregnanolone

FDA Approves Rocklatan (netarsudil and latanoprost ophthalmic solution) for the Reduction of Intraocular Pressure in Open-Angle Glaucoma or Ocular Hypertension

In continuation of my update on latanoprost 

Aerie Pharmaceuticals, Inc. (NASDAQ:AERI) (Aerie or the Company), an ophthalmic pharmaceutical company focused on the discovery, development and commercialization of first-in-class therapies for the treatment of patients with open-angle glaucoma, retinal diseases and other diseases of the eye,   announced that the U.S. Food and Drug Administration (FDA) has approved Rocklatan (netarsudil and latanoprost ophthalmic solution) 0.02%/0.005% to reduce elevated intraocular pressure (IOP) in patients with open-angle glaucoma or ocular hypertension. 

Rocklatan is a once-daily eye drop that is a fixed-dose combination of latanoprost, the most widely-prescribed prostaglandin analog (PGA), and netarsudil, the active ingredient in Rhopressa (netarsudil ophthalmic solution) 0.02%, a first-in-class Rho kinase (ROCK) inhibitor specifically designed to target the trabecular meshwork (the eye’s principal drainage pathway). The diseased trabecular meshwork is considered to be the main cause of elevated IOP in open-angle glaucoma and ocular hypertension. Rhopressa® works by restoring outflow through the trabecular meshwork, while latanoprost increases fluid outflow through a secondary mechanism known as the uveoscleral pathway.

Aerie launched Rhopressa® in the United States in April 2018. The Company plans to launch Rocklatan™ in the United States in the second quarter of 2019.

“We are in the unique position of receiving FDA approval on a second glaucoma treatment less than a year from the U.S. launch of Rhopressa®,” said Vicente Anido, Jr., Ph.D., chairman and chief executive officer at Aerie. “Together, Rocklatan™ and Rhopressa® give us a broad therapeutic franchise, based on our ROCK inhibitor netarsudil, that addresses many of the needs of clinicians and patients in a wide variety of treatment settings. Our existing salesforce, which has been calling on U.S. eye-care professionals since last May, is very well positioned to introduce Rocklatan™ to these doctors and help them understand the clinical utility of both products in the care of their patients with glaucoma. We have also been working diligently on securing favorable reimbursement for our products, with Rhopressa® now enjoying broad commercial and Medicare Part D coverage, and Rocklatan™ already under review by major payers.”

The FDA approval of Rocklatan is based on data from two Phase 3 registration trials, MERCURY 1 and MERCURY 2. In these studies, Rocklatan™ achieved its primary 90-day efficacy endpoint as well as positive 12-month safety and efficacy results, demonstrating statistically superior IOP reduction over latanoprost and netarsudil at every measured time point. More than 60% of patients taking Rocklatan™ in the two MERCURY studies achieved an IOP reduction of 30% or more, a frequency that was nearly twice that achieved by participants taking latanoprost alone. Rocklatan™ also helped more patients get to low target pressures. Nearly twice as many patients taking Rocklatan™ reached 16 mmHg or lower and nearly three times as many reached 14 mmHg or lower compared to latanoprost.

In the two MERCURY studies, Rocklatan treatment was associated with generally mild and tolerable ocular adverse events, with minimal systemic side effects. The most common ocular adverse event in controlled clinical studies with Rocklatan™ was conjunctival hyperemia. Ninety percent of patients who experienced hyperemia reported it as mild and 5% discontinued because of it. Other common ocular adverse effects reported in the studies include instillation site pain, corneal verticillata and conjunctival hemorrhage.

About Rocklatan

Indications and Usage

Rocklatan (netarsudil and latanoprost ophthalmic solution) 0.02%/0.005% is indicated for the reduction of elevated intraocular pressure (IOP) in patients with open-angle glaucoma or ocular hypertension.

Select Important Safety Information

Although not observed in the two MERCURY studies, latanoprost has been reported to cause changes to pigmented tissues, including pigmentation of the iris, periorbital tissue (eyelid) and eyelashes. Iris pigmentation is likely to be permanent. Latanoprost has also been associated with gradual changes to eyelashes including increased length, thickness and number of lashes. These changes are usually reversible.

Ewf https://www.drugbank.ca/drugs/DB00654

https://www.biospace.com/article/fda-approves-aerie-pharma-s-glaucoma-combo-eye-drops/