Pictured: A scientist works behind an FDA sign/Taylor Tieden for BioSpace

The FDA approved 55 new drugs and 34 cell and gene therapies in 2023.But its not always good news that companies have to deliver to their stakeholders; the year also had its fair share of Complete Response Letters.

As we embark on 2024,BioSpaceis committed to keeping you up-to-date on all the FDAs actions in thisFDA Decision Tracker.

June 10

Product: Ipsen and Genfits Iqirvo

Indication: Primary biliary cholangitis

Monday, the FDA approved the first new drug in nearly a decade for primary biliary cholangitis: Ipsen and Genfits Iqirvo. A rare liver disease, PBC affects around 100,000 people in the U.S. and can lead to liver failure.

Iqirvo is intended to be used in combination with ursodeoxycholic acid (UDCA) in adult patients who have an inadequate response to UDCA, or as monotherapy in patients unable to tolerate UDCA.

The companies won accelerated approval for Iqirvo based on a reduction of alkaline phosphatase, a biochemical marker often used as a surrogate endpoint in PBC studies. Treatment with the drug demonstrated statistically significant improvements in biochemical response compared to UDCA alone, Christelle Huguet, executive vice president and head of research and development at Ipsen, said in a press release. An improvement in survival or prevention of liver decompensation events has not yet been shown, and the companies may need to run a confirmatory trial to verify Iqirvos clinical benefit.

June 10

Product: Almiralls Klisyri

Indication: Actinic keratosis

Dermatology company Almirall secured expanded approval of Klisyri for larger actinic keratosis-affected areas of the face or scalp. Klisyri can now be used to treat lesions up to 100 cm2 caused by the pre-cancerous dermatological condition, after safety and tolerability profiles were consistent with original pivotal trial results.

The new authorization for Klisyri, a microtubule inhibitor ointment, increases dosing for surface area treatment from up to 25 cm2 to up to 100 cm2, according to the companys press release.

In the same press release, Almirall Chief Scientific Officer Karl Ziegelbauer called the expanded approval a significant step forward for both patients and treating dermatologists, adding that the latter are looking for ways to treat the entire affected area to help prevent further lesion progression.

June 7

Product: Gerons Rytelo

Indication: Myelodysplastic syndromes

Geron Corporation kicked off the weekend on a high note as the FDA approval of its telomerase inhibitor Rytelo for myelodysplastic syndromes (MDS)a group of blood cancerssent the companys stock soaring more than 30%. Rytelo is specifically approved for MDS patients with transfusion-dependent anemia who do not respond to or are ineligible for the standard-of-care treatment, erythropoiesis-stimulating agents.

The approvalGerons first after 34 years in businesswas supported by data from the Phase III IMerge trial, in which patients on Rytelo had significantly higher rates of red blood cell transfusion independence over placebo for at least 24 weeks28% in the treatment arm versus 3% on placebo. For those who responded, this was sustained for a median of 1.5 years.

June 7

Product: GSKs Arexvy

Indication: Respiratory syncytial virus

People ages 5059 at an increased risk of severe outcomes from respiratory syncytial virus have a new preventative option after the FDA greenlit GSKs RSV vaccine Arexvy for this subgroup on Friday. Arexvy is indicated for the prevention of lower respiratory tract disease associated with RSV.

Fridays label expansionwhich was backed by strong immunogenicity and safety data in this populationextends the market reach for Arexvy, which became the first vaccine for RSV in May 2023, at that point intended for adults 60 and above.

GSK is also evaluating the vaccine for use in people 18-49 at increased risk of severe disease, and immunocompromised patients 18 and older.

May 31

Product: Modernas mRESVIA

Indication: Respiratory syncytial virus

Moderna has a second product on the market after the FDA approved mRESVIAformerly mRNA-1345to protect adults 60 years and older fromrespiratory syncytial virus (RSV). In a press release, Moderna CEO Stphane Bancel touted the strength and versatility of the companys mRNA platform, adding that the approval also marks the first time an mRNA vaccine has been approved for a disease other than COVID-19.

mRESVIA won approval based on the Phase III ConquerRSV trial, a global study of around 37,000 adults aged 60 or older in 22 countries, in which it displayed an efficacy rate of 83.7% against RSV lower respiratory tract disease. No serious safety concerns were identified in the trial.

May 30

Product: BMSs Breyanzi

Indication: Mantle Cell Lymphoma

After winning approval earlier this month in follicular lymphoma, Bristol Myers Squibbs Breyanzi got the FDA nod for another indication on Thursday: relapsed or refractory mantle cell lymphoma (MCL). Specifically, Breyanzi is approved for patients with MCL who have received at least two prior lines of systemic therapy, including a Bruton tyrosine kinase inhibitor.

The approval is backed by the results of the MCL cohort of TRANSCEND NHL 001, where treatment with Breyanzi elicited a 67.6% complete response rate in the target patient population.

Thursdays approval marks the fourth indication for Breyanzi, making it the CAR T cell therapy available to treat the broadest array of B-cell malignancies, according to BMSs press release.

May 29

Product: Eli Lillys Retevmo

Indication: RET-altered pediatric cancers

Eli Lilly won accelerated approval Wednesday for Retevmo to treat pediatric patients two years and older with RET-positive thyroid cancers and other solid tumors that carry the mutation. Retevmo is the first drug in the class available for children under 12 years of age, Pharmaphorum reported.

Retevmo is specifically indicated for advanced or metastatic medullary thyroid cancer with a RET mutation, advanced or metastatic thyroid cancer with a RET gene fusion untreatable with radioactive iodine therapy, and locally advanced or metastatic solid tumors with a RET gene fusion that have progressed after prior systemic treatment or have no treatment options, according to the publication.

The new approval for Retevmo, which was previously authorized to treat patients 12 and older with RET-positive thyroid cancers, is based on a single-arm study that showed an overall response rate of 48%, with a median duration of response not reached after 12 months of follow-up.

May 29

Product: Tris Pharmas Onyda XR

Indication: Attention deficit hyperactivity disorder

Wednesday, the FDA greenlit Tris Pharmas Onyda XR as the first non-stimulant medication for attention deficit hyperactivity disorder (ADHD) with a liquid formulation and nighttime dosing, according to the company. Onyda XR is a reformulation of clonidine hydrochloride, which was first approved by the FDA in 1974 to treat high blood pressure. Clonidine was approved for ADHD in 2010 under the brand name Kapvay, which is owned by Shionogi.

Onyda XR leverages Tris LiquiXR platform, producing a smooth, extended-release profile, per the biotech.

Approved for patients six years and older, Tris expects to have Onyda XR available in U.S. pharmacies by the second half of 2024.

May 29

Product: Tevas Austedo XR

Indication: Tardive dyskinesia and Huntingtons disease chorea

People with tardive dyskinesia and Huntingtons disease chorea have a streamlined treatment option after the FDA approved a new one-pill-a-day version of Tevas Austedo XR. The newly approved formulation offers more flexibility with the most once-daily doses of any vesicular monoamine transporter 2 (VMAT2) inhibitor, for these conditions, according to Tevas press release. Austedo XR comes in four tablet strengths: 30, 36, 42 and 48 mg.

Austedo XR, a once-daily extended-release formulation, was first approved in February 2023.

May 28

Product: Amgens Bkemv

Indication: Paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome

AstraZenecas rare disease drug Soliris now has a biosimilar on the market after the FDA greenlit Amgens Bkemv to treat paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). Bkemv was granted the FDAs interchangeability designation, which allows it to be used in place of the branded reference product without needing to change the prescription.

Like Soliris, Bkemv carries a boxed warning for meningococcal infections, which according to its label can be serious and life-threatening. Thus, it is only available through a restricted Risk Evaluation and Mitigation Strategies program.

May 16

Product: Amgens Imdelltra

Indication: Small cell lung cancer

Amgen secured approval Thursday for its first-in-class bi-specific T-cell engager, Imdelltra, for extensive-stage small cell lung cancer (SCLC). With the FDA nod, Imdelltra becomes the first bispecific T-cell engager therapy for advanced SCLC.

The accelerated approval was based on a Phase II study of 99 patients in the target population, where Imdelltra led to an overall response rate of 40% and a median duration of response of 9.7 months. Imdelltras label contains a boxed warning for serious or life-threatening cytokine release syndrome and neurologic toxicity, including immune effector cell-associated neurotoxicity syndrome, according to the FDAs press release.

May 15

Product: BMSs Breyanzi

Indication: Follicular lymphoma

Bristol Myers Squibbs Breyanzi is now approved for the treatment of relapsed or refractory follicular lymphoma after the FDA granted a label expansion under its accelerated approval pathway. The approval was backed by data from the Phase II TRANSCEND FL study in which treatment with the CAR T cell therapy led to a 95.7% overall response rate, with a complete response rate of 73.4%.

Breyanzi, which first won approval in February 2021 for relapsed or refractory large B cell lymphoma, is also authorized to treat small lymphocytic leukemia and chronic lymphocytic leukemia. By May 31, the FDA is expected to decide whether to grant approval for the therapy in refractory mantle cell lymphoma.

May 14

Product: Dynavaxs Heplisav-B

Indication: Hepatitis B patients undergoing hemodialysis

The FDA declined to approve the supplemental Biologics License Application for Dynavax Technologies hepatitis B vaccine in patients undergoing hemodialysis, deeming the safety and efficacy data submitted by the company insufficient.

In its Complete Response Letter, the regulator said the data was insufficient because a third-party clinical site operator destroyed data source documents for about half of the subjects enrolled in the vaccines trial, according to Reuters.

While the vaccine, Heplisav-B, initially won approval for the prevention of hepatitis B in 2017, its path to the market was rocky, with two previous rejections in 2013 and 2016 for unresolved safety concerns, per Reuters.

May 1

Product: Boehringer Ingelheims Cyltezo

Indication: Rheumatoid arthritis, Crohns disease, ulcerative colitis and more

Theres another new biosimilar option to AbbVies blockbuster antirheumatic Humira. Wednesday, the FDA greenlit a high-concentration and citrate-free version of Boehringer Ingelheims Cyltezo, which was originally approved in October 2021. The newly approved dose is 100 mg/mL and is sold at a 5% discount to the branded reference product.

Cyltezo is indicated for all the same conditions as Humira, including moderate-to-severe rheumatoid arthritis, Crohns disease and ulcerative colitis. Wednesdays approval is backed by data from the Phase I VOLTAIRE-HCLF study, which compared the bioavailability of the high- and low-concentration (50 mg/mL) formulation of Cyltezo in 200 healthy volunteers.

April 30

Product: Neurocrine Biosciences Ingrezza

Indication: Huntingtons disease

A more convenient version of Neurocrine Biosciences Ingrezza will be hitting the market to treat tardive dyskinesia and chorea in Huntingtons disease after the FDA closed out April by approving a sprinkle capsule formulation of the drug.

Like the original capsule version, which was approved in 2017 for tardive dyskinesia and in 2023 for chorea in Huntingtons, Ingrezzas sprinkle formulation comes in 40-mg, 60-mg and 80-mg doses but is designed to be opened and sprinkled on soft foods. This format could be more accessible for patients who have trouble swallowing whole capsules, according to the Neurocrines announcement, which also noted that a survey of Huntingtons patients with chorea and their caregivers showed that 62% had difficulty swallowing due to their involuntary movements.

April 29

Product: Pfizer and Genmabs Tivdak

Indication: Cervical cancer

The FDA has converted the accelerated approval of Pfizer and Genmabs Tivdak into a full nod for recurrent or metastatic cervical cancer that has progressed on or after chemotherapy.

The antibody-drug conjugate (ADC), which was originally developed under a partnership between Seagen and Genmab, was granted accelerated approval in September 2021 based on a 24% objective response rate seen in the Phase II innovaTV 204 trial.

In the Phase III innovaTV 301 study, which enrolled more than 500 patients, Tivdak significantly boosted survival versus chemotherapy. An October 2023 readout showed the ADC cut the risk of death by 30% in patients with recurrent or metastatic cervical cancer; it also reduced the risk of death or worsening disease by 33% versus chemotherapy. No new safety signals were observed.

April 29

Product: X4 Pharmaceuticals Xolremdi

Indication: WHIM Syndrome

The FDAapprovedX4 Pharmaceuticals Xolremdi Monday as the first targeted treatment for WHIM syndrome, an ultra-rare immunodeficiency disease named for its four characteristics: warts, hypogammaglobulinemia, infections and myelokathexis.

Myelokathexis is a congential disorder of the white blood cells, and Xolremdi, an oral CXCR4 antagonist, is designed to mobilize white blood cells such as neutrophils, lymphocytes and monocytes from the bone marrow into the blood to improve immune deficiencies.

In the Phase III 4WHIM trial, Xolremdi showed a 60% reduction in annualized infection rate compared to placebo; trial participants had less than one infection per year compared with 4.5 for the placebo group. Patients saw an even greater reduction with additional time on treatment.

Its an exciting time for personalized medicine, and I think WHIM is going to be a poster child for rare diseases and the ability where were at now in modern medicine to design therapies to treat underlying genetic disorders, Teresa Tarrant, an associate professor at Duke Universitys School of Medicine and lead investigator of the 4WHIM trial, told BioSpace prior to Xolremdis approval.

April 26

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FDA Greenlights First Drug in Nearly a Decade for Rare Liver Disease - BioSpace