market research

FDA Approved Drugs: August, 2023

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Izervay (avacincaptad pegol sodium)

FDA approved Izervay, a complement C5 inhibitor, for the treatment of geographic atrophy secondary to age-related macular degeneration, according to a press release from Astellas Pharma.

The approval was based on the data from the GATHER1 and GATHER2 phase 3 clinical trials in which monthly 2 mg injections of Izervay (avacincaptad pegol intravitreal solution) showed a statistically significant reduction (P < .01) in the rate of geographic atrophy growth at 12 months. The slowing in disease progression was seen as early as 6 months, while up to a 35% reduction in progression occurred in the first year of treatment.

Talvey (talquetamab-tgvs)

FDA granted accelerated approval of Talvey (talquetamab-tgvs), a first-in-class bispecific antibody for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 antibody.

Talvey is a bispecific T-cell engaging antibody that binds to the CD3 receptor on the surface of T cells and G protein-coupled receptor class C group 5 member D (GPRC5D) expressed on the surface of multiple myeloma cells, non-malignant plasma cells and healthy tissue such as epithelial cells in keratinized tissues of the skin and tongue.

Elrexfio (elranatamab-bcmm)

FDA granted accelerated approval to elranatamab-bcmm (Elrexfio, Pfizer, Inc.), a bispecific B-cell maturation antigen (BCMA)-directed CD3 T-cell engager, for adults with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody.

Sohonos (palovarotene)

FDA approved Sohonos (palovarotene) capsules as a retinoid indicated for the reduction in volume of new heterotopic ossification in adults and pediatric patients aged 8 years and older for females and 10 years and older for males with fibrodysplasia ossificans progressiva (FOP).

The FDA approval was based on the pivotal efficacy and safety data from the Phase 3 MOVE trial, the first and largest multicenter, open-label trial in adult and pediatric patients.

Veopoz (pozelimab-bbfg)

FDA approved Veopoz (pozelimab-bbfg) for the treatment of adult and pediatric patients 1 year of age and older with CHAPLE disease, also known as CD55-deficient protein-losing enteropathy. Veopoz is the first and only treatment indicated specifically for CHAPLE. Veopoz, a fully human monoclonal antibody, is designed to target complement factor C5, a protein involved in complement system activation.

The FDA approval was based on results from a Phase 2/3 open-label trial that investigated the efficacy and safety of pozelimab in 10 patients aged 3 to 19 (median of 8.5 years).

Eylea HD (aflibercept)

FDA approved EYLEA HD (aflibercept) Injection 8 mg for the treatment of patients with wet age-related macular degeneration (wAMD), diabetic macular edema (DME) and diabetic retinopathy (DR). This is the first and only treatment approved in wAMD and DME for immediate dosing at 8-week and up to 16-week intervals following three initial monthly doses

Approval was based on the pivotal PULSAR and PHOTON trials in which EYLEA HD demonstrated clinically equivalent vision gains to EYLEA (aflibercept) Injection 2 mg that were maintained with fewer injections.

Tyruko (natalizumab-sztn)

FDA approved biosimilar Tyruko (natalizumab-sztn), developed by Polpharma Biologics. Tyruko is approved to treat all indications covered by the reference medicine and is the first and only FDA-approved biosimilar for relapsing forms of multiple sclerosis (MS).

Sandoz entered into a global commercialization agreement for Tyruko with Polpharma Biologics in 2019. Under this agreement, Polpharma Biologics will maintain responsibility for development, manufacturing and supply of the active substance in Tyruko. Through an exclusive global license, Sandoz has the rights to commercialize and distribute it in all markets.

FDA Approved Drugs: October, 2022

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Imjudo (tremelimumab)

AstraZeneca’s Imjudo (tremelimumab) was approved by FDA in combination with Imfinzi (durvalumab) for treating unresectable liver cancer. HCC is the most common type of liver cancer.

Imjudo (tremelimumab) is a human monoclonal antibody that targets the activity of cytotoxic T-lymphocyte-associated protein 4 (CTLA-4). Imjudo blocks the activity of CTLA-4, contributing to T-cell activation, priming the immune response to cancer and fostering cancer cell death.

The approval was based on positive results from a Phase III trial (HIMALAYA), which demonstrated that patients treated with the combination of Imjudo and Imfinzi experienced a 22% reduction in the risk of death compared to sorafenib, an oral kinase inhibitor used to treat late-stage liver cancer. The results of the Phase III trial also showed evidence that the combination therapy allowed an estimated 31% of patients to live three years after administration, compared to 20% of sorafenib-treated patients, who showed the same longevity.

Beyond HIMALAYA, Imjudo is being tested in combination with Imfinzi across multiple tumour types including locoregional HCC (EMERALD-3), SCLC (ADRIATIC) and bladder cancer (VOLGA and NILE).

Imjudo is also under review by global regulatory authorities in combination with Imfinzi and chemotherapy in 1st-line metastatic NSCLC based on the results of the POSEIDON Phase III trial, which showed the addition of a short course of Imjudo to Imfinzi plus chemotherapy improved both overall and progression-free survival compared to chemotherapy alone.

Tecvayli (teclistamab-cqyv)

Janssen’s Tecvayli (teclistamab-cqyv) is a bispecific B-cell maturation antigen (BCMA)-directed CD3 T-cell engager indicated for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent and an anti-CD38 monoclonal antibody.

  • Multiple myeloma is a cancer of the plasma cells in the bone marrow. Plasma cells normally make antibodies which fight infection, but when the plasma cells become malignant and develop into multiple myeloma, the myeloma cells proliferate and replace normal cells in the bone marrow.
  • Tecvayli targets both BCMA (B-cell maturation antigen) and CD3, the T-cell receptor. BCMA is expressed at high levels on multiple myeloma cells. Tecvayli works by redirecting CD3-positive T-cells to BCMA-expressing myeloma cells to induce the killing of the cancer cells.
  • Warnings and precautions associated with Tecvayli include hepatotoxicity, infections, neutropenia, hypersensitivity and other administration reactions, and embryo-fetal toxicity.
  • Common adverse reactions observed in clinical trials include cytokine release syndrome, neutropenia, and anemia. Infections were frequent with the most common being upper respiratory tract infections and pneumonia. Hypogammaglobinemia and neurotoxic events were also observed in some patients.

FDA Approved Drugs: May & June, 2022

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Amvuttra (Alnylam Pharma)

Vutrisiran, to be marketed under the brand name Amvuttra, has won an FDA approval to treat hereditary transthyretin amyloidosis (hATTR) polyneuropathy in adults. In the phase III HELIOS-A study, Amvuttra helped ATTR polyneuropathy patients achieve an average 2.2-point improvement on a modified neuropathy impairment score after nine months; half of the patients experienced some level of improvement. In the same study, Onpattro takers got 1.4 points better. By comparison, patients who took placebo in a historical Onpattro clinical trial called APOLLO experienced disease worsening of an average 17 points.

Voquezna (Phathom)

VoqueznaTriple Pak(vonoprazan, amoxicillin, clarithromycin) and Voquezna Dual Pak(vonoprazan, amoxicillin) is approved for the treatment of Helicobacter pylori (H. pylori) infection in adults.

The approval was based on data from a randomized, controlled, double-blind triple therapy/open-label dual therapy, phase III study HP-301, which compared the efficacy and safety of Voquezna Triple and Dual Pak with lansoprazole in combination with amoxicillin and clarithromycin in 1046 adults with H. pylori infection.

Mounjaro (Lilly)

Mounjaro is an injectable prescription medicine that is used along with diet and exercise to improve blood sugar (glucose) in adults with type 2 diabetes mellitus. Mounjaro is not for use in people with type 1 diabetes. It is not known if Mounjaro is safe and effective for use in children under 18 years of age. Mounjaro is a dual GLP-1 and GIP agonist and has demonstrated superior efficacy compared to other diabetes medications.The approval of Mounjaro was based on results from the phase III SURPASS program, which compared it to competitors such as injectable semaglutide, insulin glargine, and insulin degludec.

Vtama (Dermavant)

Vtama(tapinarof) Cream, 1% is an aryl hydrocarbon receptor agonist indicated for the topical treatment of plaque psoriasis in adults.

The approval was based on data obtained from 2 identical phase III trials that assessed the safety and efficacy of Vtama in 1025 adults (18 to 75 years of age) diagnosed with plaque psoriasis.

The primary endpoint for both studies was the hope that patients would earn a Physician Global Assessment (PGA) score of clear (0) or almost clear (1). Results from both trials showed that 36% and 40% of patients met the primary endpoint, respectively. Following 12 weeks of treatment, 73 Vtama patients achieved complete disease clearance (PGA 0).

FDA Approved Drugs: November, 2021

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Voxzogo (BioMarin Pharmaceutical)

BioMarin Pharmaceutical’s Voxzogo (vosoritide) is the first FDA approved therapy for children with achondroplasia, an inherited disorder that causes the most common form of dwarfism. With this approval, the FDA also issued a Rare Paediatric Disease Priority Review Voucher (PRV), which confers priority review to a subsequent drug application that would not otherwise qualify for priority review. Voxzogo, a C-type natriuretic peptide (CNP) analog, represents a new class of therapy, which acts as a positive regulator of the signaling pathway downstream of FGFR3 to promote endochondral bone growth.

Livtencity (Takeda)

Takeda’s Livtencity (maribavir) is the first and only treatment for adults and pediatric patients (12 years of age and older and weighing at least 35 kg) with post-transplant cytomegalovirus (CMV) infection/disease that is refractory to treatment (with or without genotypic resistance) with ganciclovir, valganciclovir, cidofovir or foscarnet. This new molecular entity targets CMV at UL97, resulting in inhibition of viral DNA replication, encapsidation, and nuclear egress. It became available for prescription on December 2, 2021.

Cytalux (On Target Laboratories)

On Target Laboratories’s Cytalux (pafolacianine) is the first targeted fluorescent imaging agent indicated as an adjunct for the intraoperative identification of malignant lesions in patients with ovarian cancer. Cytalux, administered by standard IV in as little as one hour before surgery, binds to folate receptors that are overexpressed in most epithelial ovarian cancers and illuminate intraoperatively under near-infrared light.

Cutaneous T-cell Lymphoma (CTCL): Market Size & Forecast

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Cutaneous T-cell Lymphoma (CTCL): Market Size & Forecast

Disease Overview

Cutaneous T-cell Lymphoma (CTCL) is the most common type of skin lymphoma. CTCL is rare cancer that begins in white blood cells called T cells (T lymphocytes) and these the T cells develop abnormalities that make them attack the skin. It appears as an eczema-like skin rashes and can affect widespread parts of the body.

There are different types of Cutaneous T-cell Lymphoma (CTCL). Mycosis Fungoides and Sezary syndrome are the most frequent type of Cutaneous T-cell Lymphoma (CTCL). The other forms of cutaneous lymphoma include the CD30 positive lymphoproliferative disorders (CD30+ LPDs), subcutaneous panniculitis-like T-cell lymphoma, Primary cutaneous CD4+ small/medium pleomorphic T-cell lymphoma, Primary cutaneous gamma/delta T-cell lymphoma, Extranodal natural killer/T-cell lymphoma, nasal type, and others.

The World Health Organization (WHO) and the European Organization for Research and Treatment of Cancer (EORTC) Classification

Indolent Clinical Behavior Aggressive Clinical Behavior
Mycosis fungoides Sézary syndrome  
Subcutaneous panniculitis-like T-cell lymphoma Primary cutaneous CD8+ aggressive epidermotopic T-cell lymphoma  
Primary cutaneous CD4+ small/medium pleomorphic T-Cell lymphoma   Primary cutaneous gamma/delta T-cell lymphoma  
CD30+ Lymphproliferative Disorders Primary cutaneous anaplastic large cell lymphomaLymphomatoid papulosis   Extranodal natural killer/T-cell lymphoma, nasal type  

Epidemiology

According to the CmaxInsight, the total incident cases of Cutaneous T-cell lymphoma (CTCL) were calculated to be around 9,000 in 2018 in 7MM including the United States, EU5 countries (the United Kingdom, France, Italy, Spain, and Germany) and Japan. The United States accounts for the highest incident cases of 3,430  in 2018 followed by EU5 countries, and Japan According to the National Organization for Rare Disorders (NORD), the Cutaneous T-cell lymphoma (CTCL) affects males twice as often as females. The majority of the CTCL cases are diagnosed between the age-group of 40 to 60 years. Further, it is expected that the incident cases of the CTCL would increase over the forecasted period.

Market Size

According to the Cmaxinsight, the market size of Cutaneous T-cell Lymphoma (CTCL) is growing at a CAGR of 5.54% for the forecasted period 2018-2028 in 7MM including the United States, EU5 countries (the United Kingdom, France, Italy, Spain, and Germany) and Japan. The United States accounts for the highest market share of around 40% in 2018 followed by EU5 countries, and Japan. The market is dominated by the availability of topical therapies, including corticosteroids, retinoids, different chemotherapy regimens, and others. The market is driven by rising incident cases, the expected launch of emerging therapies, technological advancement, and improving healthcare facilities.

Competitive Analysis

The market is dominated by the availability of the marketed drugs such as Istodax (Celgene Corporation), Poteligeo (Kyowa Hakko Kirin Co. Ltd), Valchlor/ Ledaga (Actelion), Uvadex (Mallinckrodt Pharmaceuticals), Targretin (Eisai Co., Ltd.), and Zolinza (Merck Sharp & Dohme Corp), and others. The expected launch of emerging therapies such as Quisinostat (Janssen), Remetinostat (Medivir AB), denileukin diftitox (Eisai Co., Ltd) and others shall have a positive impact on the market.

The companies are entering into the mergers, acquisitions, collaborations and other activities for increasing their market penetration. In January 2020, the Almirall had entered into an option agreement to acquire Bioniz Therapeutics and to establish a broad research agreement to further expand its innovative pipeline in medical dermatology. This collaboration would allow Almirall to executes its strategy to develop and expand its R&D pipeline with new treatment modalities with the objective to address highly underserved diseases within oncodermatology and immunodermatology.