Breaking News: GSK, Citius, and Harm Reduction Therapeutics Await FDA Verdict in 2023

A whirlwind of activity has been seen in the first half of 2023, as the FDA has already approved a record 28 new treatments. This bodes well for the biopharma industry, who can expect further novel treatments, including some promising first-in-class therapies, to be swiftly approved. Unfortunately, not all news has been good, as several Complete Response Letters have also been sent out on various products.

At BioSpace, we’re getting ready to launch our FDA Decision Tracker, so you can stay on top of all new FDA actions. With our comprehensive tracker, you’ll be able to stay up to date on the latest developments and keep your finger on the pulse of the industry!


July 31:

After decades without a new frontline treatment, the U.S. Food and Drug Administration (FDA) has approved GlaxoSmithKline’s Jemperli (dostarlimab-gxly) as a frontline therapy for primary advanced or recurrent endometrial cancer in patients whose cancer is mismatch repair deficient (dMMR) or microsatellite instability-high (MSI-H). Jemperli is the first immuno-oncology treatment and PD-1 inhibitor to receive FDA authorization for frontline use in this patient population, bringing hope to those suffering from the disease.

On Monday, a milestone in oncology was reached with the earlier-than-expected approval of Jemperli, an anti-PD-1 antibody. First approved in April 2021 to treat recurrent or advanced dMMR endometrial cancer, the FDA accepted a supplemental BLA in June to move the drug into the frontline setting, with a target action date originally set to September 23. However, on Monday the drug was given the green light two months ahead of schedule, presenting the promise of more effective treatments to oncology patients.

July 29:

Citius Pharmaceuticals’ ambitions to bring back Eisai’s cancer drug Ontak as a reformulated version were brought to a halt on Saturday when the U.S. Food and Drug Administration (FDA) denied its application for Lymphir (denileukin diftitox). Acquired from Dr. Reddy’s Laboratories just last September, the setback may spell out more development and approval delays for the potential lifesaving treatment.

Lymphir is an advanced two-in-one drug designed to fight relapsed or refractory Cutaneous T-Cell Lymphoma (CTCL). The protective fusion protein co-opts the body’s own IL-2 receptors to deliver its toxin payload into malignant T-cells, effectively halting their protein synthesis. By targeting immunosuppressive regulatory T-cells as well, Lymphir’s innovative one-two punch boosts the body’s own defenses, allowing it to mount a more effective response against the cancer.

Citius recently announced it had received a Complete Response Letter from the U.S. Food and Drug Administration regarding its biologics license application (BLA) for Lymphir. Thankfully, no clinical efficacy or safety issues were raised, only the need to incorporate enhanced product testing and additional controls into the BLA. Citius remains committed to working to getting Lymphir approved by the FDA.

July 28:

In a move to combat the growing opioid epidemic, the FDA has approved Harm Reduction Therapeutics’ 3-mg naloxone hydrochloride nasal spray, RiVive, as the second over-the-counter (OTC) naloxone rescue option. This approval comes just four months after the regulator greenlit Narcan, a 4-mg naloxone hydrochloride nasal spray from Emergent BioSolutions, as the first OTC naloxone product. Rapidly reversing the effects of opioid overdose, naloxone is a much-needed weapon against the tragic number of lives lost—105,000 in the U.S. in the year ending February 2023—due to this ongoing crisis.

The FDA has officially approved RiVive, a naloxone product developed to help those suffering from opioid overdoses. In celebration of this major milestone, FDA Commissioner Robert Califf expressed his commitment to making naloxone more accessible and encouraging manufacturers to continue working with the FDA to develop non-prescription tools.

July 26:

To counter the serious dangers posed by Warfarin, Octapharma has just secured approval for Balfaxar, an innovative prothrombin complex concentrate. Warfarin, a blood thinner, increases the risk of dangerous bleeding during surgery and invasive procedures, but Balfaxar is designed to quickly increase blood levels of key clotting factors and antithrombotic proteins, giving doctors and patients an urgently needed solution. Balfaxar is a lyophilized powder that is then reconstituted using a device developed by Octapharma.

Balfaxar has just passed the test of hemostatic efficacy in a Phase III study and was proved to be a non-inferior to its rival, Kcentra. Unfortunately, though, fatal and non-fatal thromboembolic complications have been reported as a side-effect of its consumption, both in clinical trials and post-marketing studies. To be on the safe side, healthcare professionals recommend to closely monitor their patients for any potential signs and risks of thromboembolic events.

Balfaxar has already seen success in Canada and the EU, and now it has received its third approval, establishing itself as the powerful octaplex it was meant to be.

July 25:

Suffenders of the eyelid disease called demodex blepharitis now have a groundbreaking treatment option! The FDA has recently approved Tarsus Pharmaceuticals’ Xdemvy. This revolutionary prescription eye drop aims to be the ultimate solution to eradicating the root cause of the condition–demodex mites burrowing in the eyelash follicles. Xdemvy is historic first therapy to be approved for the condition, offering an exciting new start for those with demodex blepharitis.

Xdemvy was granted the green light for use in the public, following two randomized, multi-center, double-masked, and vehicle-controlled studies of 833 individuals – 415 of which were treated. In each study, a remarkable improvement was seen in just 43 days! According to Tarsus, the medication was “generally safe and well-tolerated,” making it a win-win situation for everyone!

Tarsus has set its sights on a potentially game-changing market with Xdemvy; up to 25 million Americans may suffer from demodex blepharitis, representing two thirds of all blepharitis cases. Xdemvy could offer a vital solution to a health issue which effects millions of US citizens.

July 24:

The FDA has approved YCANTH, the first-of-its-kind treatment for adults and children two years and older who suffer from molluscum contagiosum. This common skin infection affects roughly 6 million people in the U.S. each year. Developed by Verrica Pharmaceuticals, YCANTH is a drug-device combination containing a GMP-controlled formulation of cantharidin, which originates from the blister beetle Cantharis vesicatoria. The solution works by producing a blister on the wart or growth, and then eventually elevating it off the skin. Now, with FDA approval, those living with molluscum contagiosum can finally find relief.

Verrica Pharmaceuticals has been granted marketing approval by the U.S. Food and Drug Administration (FDA) for YCANTH, the first and only FDA-approved topical treatment for the common pediatric skin condition molluscum contagiosum. The approval is based on the results of two Phase III double-blind, randomized, multicenter clinical trials, which demonstrated clinically and statistically significant treatment success in achieving complete clearance of all treatable lesions. YCANTH is set to become available for patients and caregivers later this year in September, and no serious adverse reactions have been reported to date. With this novel treatment, Verrica Pharmaceuticals seeks to provide relief to those suffering from molluscum contagiosum, which has long lacked effective options.

July 20:

Daiichi Sankyo’s Vanflyta (quizartinib), rejected in 2017 due to safety concerns, has finally won approval to treat patients with acute myeloid leukemia that has a FLT3-ITD gene mutation. Although the FDA backed the drug in 2019, the approval comes with a Boxed Warning for three heart disorders and only those partaking in the restricted Risk Evaluation and Mitigation Strategy (REMS) program are authorized to access the drug. Torsades de pointes, QT prolongation, and cardiac arrest may now be avoided in those suffering from this severe form of cancer.

Daiichi Sankyo recently announced the exciting results from a Phase III trial of their drug quizartinib. When combined with standard induction and consolidation chemotherapy, and continued as a single agent, this treatment regimen remarkably reduced the risk of death by 22.4% compared to chemotherapy alone. Amazingly, after 39.2 months of follow-up, quizartinib more than doubled the overall survival advantage. This breakthrough has great potential to improve treatments for certain types of leukemia.

Emergent BioSolutions has just earned a major win in the fight against bacillus anthracis with the approval of Cyfendus, a breakthrough vaccine designed to protect against anthrax. In a series of successful studies, the vaccine proved to be safe and effective in healthy adults, leading to its approval for use in combination with recommended antimicrobial drugs. Emergent had already been supplying Cyfendus to the U.S. Department of Health and Human Services under pre-Emergency Use Authorization for four years prior to now, highlighting its importance in preventing a potential outbreak.

July 17:

Parents of children at severe risk of RSV can now rest easier, with the FDA’s recent approval of Sanofi and AstraZeneca’s Beyfortus (nirsevimab). This monoclonal antibody is just the second RSV drug ever authorized for use in the U.S., offering a much-needed addition to the treatments available. Supported by data drawn form three late-stage trials, Beyfortus’ safety and efficacy have been ensured. As a result, it will soon be available to children entering their first RSV season as well as those at severe risk up to 24 months of age. Beyfortus’ arrival is well-timed, with the 2023/2024 RSV season bringing new challenges and opportunities.

July 13:

In an historic move for women’s health, the FDA has authorized Perrigo Company’s Opill (norgestrel), the first over-the-counter birth control pill. Set to launch in the first quarter of 2024, Opill will be available in drugstores, convenience stores, and grocery stores as well as online, providing more convenient access to contraception. This momentous decision was celebrated by Perrigo President and CEO Patrick Lockwood-Taylor, who stated, “Today marks a truly momentous day for women’s health nationwide.”

July 6:

The U.S. week came to an exciting end as the FDA granted a full approval to Leqembi, the first anti-amyloid antibody and disease-altering drug ever for Alzheimer’s disease! It is expected that Medicare coverage for Leqembi will be available right away, with the requirement of a patient registry to further monitor the drug’s effectiveness in treating Alzheimer’s, as verified by results from the Clarity-AD trial. This marks a milestone for Alzheimer’s research and signals hope in the fight against this debilitating disorder.

July 3:

Amneal Pharmaceuticals has unfortunately received an unexpected rejection letter from the FDA for its novel oral formulation of carbidopa/levodopa (CD/LD) for the management of Parkinson’s disease. The FDA was not able to sufficiently confirm the safety of the carbidopa component, which has led to the issuance of a Complete Response Letter and the request for additional pharmacokinetic data. Amneal plans to discuss the best path forward with the FDA to advance the treatment.


June 29:

This week, the US Food and Drug Administration made a groundbreaking announcement: the FDA approved Roctavian (valoctocogene roxaparvovec-rvox), a gene therapy developed by BioMarin, as the first treatment for adults with severe hemophilia A. Hemophilia is a rare genetic disorder that disrupts the body’s ability to produce coagulating factor VIII, making it difficult to control bleeding. Roctavian is a one-time gene therapy that utilizes an adeno-associated virus (AAV) vector to deliver a healthy genetic copy of FVIII directly to the liver. This increases FVIII in the bloodstream, and potentially reduces the risk of life-threatening bleeding episodes. This marks a monumental step forward in the quest to treat hemophilia and life-threatening bleeding.

June 28:

The U.S. Food and Drug Administration has approved the first cellular therapy for type 1 diabetes – a groundbreaking milestone in the ongoing fight against the condition. Lantidra (donislecel), developed by Chicago-based CellTrans, is created from the pancreatic islet cells of deceased donors. This innovative treatment has been shown to provide hope for adult patients unable to reach average blood glucose levels due to hypoglycemic episodes. In a clinical trial of 30 individuals, 21 were completely insulin-free for at least one year, while 11 achieved insulin-free periods lasting from one to five years. Insulin independence was maintained for over five years in 10 out of 30 patients. Despite these achievements, five did not benefit from the therapy.

In December 2014, Pfizer placed a timely bet on OPKO Health’s human growth hormone analog, leading to a lucrative jackpot when the FDA finally approved the treatment—to be marketed as Ngenla—for children with impaired growth hormone production. To secure exclusive global rights to the experimental treatment, the pharma giant paid $295 million upfront and offered up to $275 million in milestones. Though initially rejected by the FDA in January 2022, Ngenla persevered and was eventually approved by the regulator.

Despite their efforts, Eton Pharmaceuticals was dealt a second consecutive blow by the FDA, as their dehydrated alcohol injection was once again declined for the treatment of methanol poisoning. With ‘chemistry manufacturing and controls’ being the primary reason for the declining, the first complete response letter issued to Eton this March was due to pandemic-driven travel restrictions which prevented a timely inspection of the company’s European contract manufacturing site.

June 27:

The U.S. Food and Drug Administration (FDA) has just given the green light to UCB’s Rystiggo, a breakthrough, subcutaneously administered IgG4 monoclonal antibody to treat generalized myasthenia gravis, a rare muscle-wasting autoimmune disease. This is the very first treatment to be approved to treat patients who are anti-acetylcholine receptor- or anti-muscle-specific tyrosine kinase antibody-positive. Its success was proven in a Phase III trial, showing Rystiggo brought significant improvements in symptoms associated with breathing, talking, swallowing and rising from a chair. Rystiggo may now provide new hope to those suffering from this debilitating disease.

June 23:

The US Food and Drug Administration has approved Pfizer’s Litfulo (ritlecitinib) as a revolutionary treatment for patients as young as 12 years with severe alopecia areata- a devastating autoimmune disorder causing patchy or total hair loss on the scalp, face and body. Through its unique ability to inhibit Janus kinase 3 and tyrosin kinase, Litfulo works by blocking the signals of the cytokines and T-cell cytolytic activity believed to be implicated in alopecia areata, giving hope to sufferers of the condition.

June 22: 

Sarepta’s groundbreaking new gene therapy, Elevidys (formerly SRP-9001), has been approved by the FDA, marking a major milestone for children 4 to 5 years of age suffering from Duchenne Muscular Dystrophy (DMD). This is the first gene therapy to be approved for DMD, a debilitating neuromuscular disorder primarily affecting young boys. The FDA granted the therapy accelerated approval based on evidence that it increases the expression of micro-dystrophin protein. This new gene therapy offers hope for children and families with DMD and is a major step forward in treating this condition.

June 21:

Eli Lilly and Boehringer Ingleheim just broke new ground in the diabetes industry, becoming the first companies to receive FDA approval for SGLT2 inhibitors Jardiance (empagliflozin) and Synjardy (empagliflozin and metformin hydrochloride) for children 10 years and older with type 2 diabetes. Metformin had previously been the only oral drug approved for this indication, and its approval was granted back in 2000. With Jardiance and Synjardy becoming available, pediatric patients now have more options when it comes to managing their type 2 diabetes.

June 20:

Argenx have made a major breakthrough in treating generalized myasthenia gravis with Vyvgart (efgartigimod): the world’s first subcutaneous injectable for the muscle-wasting autoimmune disease. The antibody fragment effectively blocks the recycling of immunoglobulin G back into the bloodstream, inhibiting the creation of antibodies that impair the ability of acetylcholine receptors to receive signals from the nerves and the resulting muscular response. Vyvgart is a game-changer, offering 85% of gMG patients a hope of improved muscular functions.

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