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.
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 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(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).
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.
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.
Qelbree (viloxazine extended-release capsules): Supernus Pharmaceuticals, Inc.
FDA approved Qelbree (viloxazine extended-release capsules) for the treatment of attention-deficit hyperactivity disorder (ADHD) in pediatric patients 6 to 17 years of age. Qelbree represents the first novel non-stimulant treatment for ADHD. Supernus Pharmaceuticalsplans to make Qelbree available in the United States in Q2 2021.
Attention deficit hyperactivity disorder (ADHD) is a mental health disorder that can cause above-normal levels of hyperactive and impulsive behaviors. People with ADHD may also have trouble focusing their attention on a single task or sitting still for long periods of time.
The approval of Qelbree is based on the data from an extensive development program consisting of four Phase III clinical trials that studied more than 1000 pediatric patients from the age of 6 to 17 years. In December 2020, the Company announced positive results from a Phase III trial in adult patients with ADHD and plans to submit a supplemental New Drug Application to the FDA for Qelbree in adults in the second half of 2021.
Nextstellis (Drospirenone and Estetrol Tablets): Mayne Pharma
FDA approved Nextstellis (also trade name: Estelle), (3 mg drospirenone [DRSP] and 14.2 mg estetrol [E4] tablets), for the prevention of pregnancy. Nextstellis is the first and only contraceptive pill containing E4, a naturally produced estrogen during pregnancy, which can now be made from a plant source. Mayne Pharma anticipates the commercial launch of Nextstellisby at the end of June 2021.
Nearly 10 million American women use short-acting combination contraceptives (estrogen and progestin). Of these contraceptives, more than 99% contain ethinylestradiol (EE), a synthetic estrogen that binds widely to all estrogen receptors in the body.
The approval is based on the two-phase III clinical studies conducted in 3,725 women, Nextstelliswas shown both safe and effective and met its primary efficacy endpoint of pregnancy prevention while also meeting a variety of secondary endpoints that demonstrated favorable cycle control, bleeding control, safety, and tolerability.
Nextstellis was developed by a Belgian biotech company Mithra Pharmaceuticals. Mithra has signed 10 licensing deals for Estelle with a number of leading women’s health companies covering United States, Europe, Japan, South Korea, ASEAN, Russia, Brazil, Canada, Middle East, North Africa, Southern Africa and Australia. Mayne Pharma has a 20-year exclusive license and supply agreement in the United States and Australia for Nextstellis.
In May 2020, Mithra has entered into an exclusive 20-year license and supply agreement with Mayne Pharma for the commercialization of its combined oral contraceptive E4/DRSP (Estelle) in Australia.
In October 2019, Mithrahas signed a License and Supply Agreement (LSA) with Mayne Pharma Group for an exclusive license to commercialize Estelle in the US.
As a result of receiving FDA approval for Nextstellis, Mayne Pharma will pay Mithra US$11m in cash and issue 85.8m ordinary Mayne Pharma shares.
Vitiligo is a chronic disorder that causes areas of skin to lose color. When skin cells that make color are attacked and destroyed, the skin turns a milky-white color. Various factors such as autoimmune disease, genetic factors, & neurogenic factors lead to the development of Vitiligo. The main symptom associated with vitiligo is the loss of natural color. The patches can show up on any part of your body and can affect the skin, which gets milky-white patches, usually on the hands, feet, arms, and face, hair, which can turn white on the scalp, eyebrow, eyelash, and beard & the inside of your mouth or nose. People with vitiligo can also get problems with the eyes and ears. In addition, people with the disorder may worry about how their skin looks, which can affect general well-being.
Vitiligo is the most common skin depigmentation disorder with an average prevalence estimated to be between 0.5% to 2% of the global population. An estimated 65-95 million people of all ages, sexes, and ethnicities worldwide suffer from vitiligo, including approximately 2.4 million people in the United States. The majority (70–80%) of patients with vitiligo experience disease onset before age 30. Approximately 30% have childhood-onset vitiligo before age 12.
Currently, there are no FDA-approved therapies for vitiligo. Dermatologists typically prescribe topical corticosteroids, topical calcineurin inhibitors, and/or phototherapy for the treatment of vitiligo. Therefore, pharmaceutical companies such as Incyte Corporation, Dermavant Sciences GmbH, Aclaris Therapeutics, Inc. Amgen and other companies are developing novel therapies for the treatment of Vitiligo.
Incyte Corporation (Ruxolitinib/INCB018424)
Ruxolitinib is a Janus kinase (JAK1/JAK2) inhibitor discovered by Incyte scientists. Overactive signaling through the JAK-STAT pathway has been associated with many types of disease, including a group of rare blood cancers called myeloproliferative neoplasms (MPNs), as well also used for the treatment of Vitiligo. Ruxolitinib is currently under the clinical trial of Phase III. The purpose of this study is to evaluate the efficacy and safety of ruxolitinib cream in adolescent and adult participants with non-segmental vitiligo for whom total body involved vitiligo area (facial and nonfacial) does not exceed 10% body surface area (BSA).
Cerdulatinib is a dual inhibitor of the Janus kinase (JAK) and spleen tyrosine kinase (Syk) pathways, which Dermavant is evaluating as a differentiated topical treatment option for vitiligo and other inflammatory skin conditions such as atopic dermatitis. The Phase IIa study is a multi-center, randomized, double-blind, vehicle-controlled study to assess the safety, tolerability, and systemic exposure of cerdulatinib gel 0.37%, dosed twice daily for six weeks versus vehicle in 30 adult patients aged 18-70 years diagnosed with vitiligo. The primary endpoints of the study are the safety and tolerability of topical administration of cerdulatinib gel 0.37% in adult subjects with vitiligo assessed by frequency, duration, and severity of adverse events (local and systemic), vital signs, laboratory values, and local tolerability scale (LTS) scores.
Aclaris Therapeutics, Inc. (ATI-50002/ATI-502)
ATI-502 (AA-201 Topical), is an investigational topical Janus Kinase (JAK) 1/3 inhibitor, developed by aclaris therapeutics used to treat Vitiligo. Currently, ATI-502 is evaluated under the clinical trial study of Phase II. This is a multicenter, open-label study designed to evaluate the safety, tolerability, and efficacy of ATI-50002 Topical Solution 0.46% in subjects with non-segmental facial vitiligo. Subjects would be required to have a clinical diagnosis of non-segmental facial vitiligo affecting at least 0.25% of total body surface area (TBSA) (excluding upper and lower eyelids, mucosal lip areas, and forehead and chin areas covered by the stereotactic positioning device for photography) with at least one area of the face with normal pigmentation. Twenty-four eligible subjects would receive ATI-50002 Topical Solution, 0.46%, BID for 24 weeks.
PF-06651600 is an oral JAK3 inhibitor used to treat Vitiligo. The JAK pathways are believed to play an important role in inflammatory processes as they are involved in signaling for over 50 cytokines and growth factors, many of which drive immune-mediated conditions. JAK inhibition may offer patients with these conditions a potential new advanced treatment option. PF-06651600 initiated a Phase II clinical trial study. This is a randomized, double-blind, parallel-group, multicenter study with an extension period. The study would have a maximum duration of approximately 60 weeks. This includes an up to 4 weeks Screening Period, a 24-week dose-ranging period, an up to 24 week extension period and an 8 week Follow up Period.
Amgen (AMG 714)
AMG 714 (PRV-015) is a human immunoglobulin monoclonal antibody that binds to IL-15 developed by amgen used to treat Vitiligo. Currently, AMG 714 is evaluated under a clinical trial study. The Phase IIa, double-blind, placebo-controlled, multi-center, proof of concept trial of AMG 714 for the treatment of vitiligo. Participants would be randomized 2:1 to receive AMG 714 or placebo for AMG714. The random assignment would be stratified by active versus stable vitiligo.