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Press Releases

Take a look at a selection of our recent media coverage:

Trofinetide approved for Rett Syndrome from two years of age

15th March 2023

Trofinetide is approved for treatment of the rare, neuro-developmental disorder Rett Syndrome in patients from two years of age

Acadia Pharmaceuticals announced that it has received approval for trofinetide as a treatment for adult and paediatric patients with Rett Syndrome from two years of age.

Rett syndrome is described as a neuro-developmental disorder characterised by typical early growth and development followed by a slowing of that development, loss of functional use of the hands, distinctive hand movements, slowed brain and head growth, problems with walking, seizures, and intellectual disability. The condition primarily affects females resulting in severe cognitive and physical disabilities and is estimated to affect about 1 in 12,000 girls and is only rarely seen in boys. Virtually all patients with Rett syndrome have one of > 300 distinct loss-of-function mutations in the MECP2 gene on the X chromosome, which encodes methyl-CpG binding protein-2, an essential transcriptional regulator in the brain required for normal neurodevelopment.

Before trofinetide, which is given orally, there were no recognised treatments for Rett syndrome and the drug is believed to work by normalising aberrant neural function resulting from MECP2 mutations. In an exploratory phase 2, multicentre, double-blind, placebo-controlled, dose-escalation study, trofinetide was found to provide a clinically meaningful improvement for patients with the syndrome. The approval by the FDA was based on the findings from LAVENDER, a phase 3 study of trofinetide for Rett syndrome.

According to the press release, LAVENDER evaluated the efficacy and safety of trofinetide compared to placebo in 187 female patients with Rett syndrome, five to 20 years of age. In the study, treatment with trofinetide produced a statistically significant improvement (compared to placebo) on both co-primary efficacy endpoints, measured by the change from baseline, in Rett Syndrome Behaviour Questionnaire (RSBQ) total score (p = 0.018), which is a caregiver assessment on a range of disease symptoms, and the Clinical Global Impression-Improvement (CGI-I) scale score (p = 0.003) at week 12, which represents a physician-based assessment. In the study, the most common side effects were diarrhoea (82%) and vomiting (29%).

The drug is likely to be available in the US from April 2023 although the press release provides no information on its release elsewhere.

Zavegepant nasal spray approved for acute migraine

In a press release, Pfizer has announced that zavegepant has become the first calcitonin gene-related peptide receptor antagonist nasal spray to receive approval by the FDA for the management of acute migraine.

According to a study using data from 2016, it was estimated that globally, migraine affects some 1·04 billion people and caused 45·1 million years of life lived with disability. Although there was a breakthrough in treatment of migraine during the early 1990’s with the introduction of triptans, it later emerged that the calcitonin-gene-related peptide (CGRP) pathway was important in the pathophysiology of migraine. While first generation drugs targeting this pathway, known as ‘gepants’ were effective, hepatotoxicity halted further development but did lead to new generation of gepants, which have been shown to be safe, efficacious and well tolerated.

Zavegepant clinical studies

Zavegepant nasal spray is a third generation gepant and the first, non-oral gepant. In a randomised, dose-ranging, placebo-controlled, phase 2/3 trial in adults aged ≥18 years with migraine, single doses of 10 or 20 mg, of the drug were shown to be effective for the acute treatment of migraine and with a favourable safety profile. Further data on the efficacy of zavegepant and which formed the basis for the approval, came from a double-blind, randomised, placebo-controlled phase 3 trial in adults with a history of two to eight moderate or severe migraine attacks per month. The results showed that among those assigned to zavegepant, two hours after the treatment dose, a statistically significant higher proportion of zavegepant patients had freedom from pain and freedom from their most bothersome symptom. In addition, twice as many patients (16% vs 8%) given zavegepant reported pain relief after only 15 minutes. There were also significant differences (compared to placebo) for 13 of the 17 pre-specified secondary outcomes.

The trial also found that the drug was well tolerated with the most common adverse reactions reported in at least 2% of patients and at a frequency greater than placebo, were taste disorders (includes dysgeusia and ageusia), nausea, nasal discomfort and vomiting.

First drug receives FDA approval for ultra rare neurodegenerative disorder

6th March 2023

Omaveloxolone is the first drug to receive FDA approval for the progressive genetic neurodegenerative disorder, Friedreich’s ataxia

According to a press release by the manufacturer, Reata, omaveloxolone (brand name SKYCLARYS™) has become the first treatment to be approved for the treatment of Friedreich’s ataxia, an ultra-rare, genetic, progressive neurological disorder.

Friedreich’s ataxia represents an inherited autosomal recessive disease, that affects the nervous system, producing a progressive ataxia, weakness and sensory deficits. The disease has a median onset age of 10 to 15 years and affects approximately 1 in 50,000 people worldwide. Friedreich’s ataxia is due to a lack of function in a gene responsible for the production of frataxin, a protein involved in mitochondrial iron homeostasis. During conditions of cellular oxidative stress, there is activation of the transcription factor, NF-E2-related factor and which triggers a cellular antioxidant response, through induction of antioxidant response element driven genes. However, it appears that a lack of frataxin impairs NF-E2 signalling and this is though a contributory factor for the neuro-degeneration seen in Friedreich’s ataxia. Omaveloxolone has been shown to induce NF-E2 and thus protect cells from oxidative stress.

Omaveloxolone clinical efficacy

An initial dosing ranging study identified that a dose of 160 mg/day appeared to improve neurological function. Following on from this, a phase 2 randomised, placebo-controlled trial with 103 patients, demonstrated that omaveloxolone 150 mg daily, significantly improved the modified Friedreich’s Ataxia Rating Scale (mFARS) score after 48 weeks compared to placebo (p = 0.014). In an open label extension trial, after 72 weeks, the response to omaveloxolone compared to placebo was maintained.

Following the FDA approval, the company has created the Reata Education, Access, and Care Helpline (REACH), an integrated specialty pharmacy and patient services program, designed to help eligible patients access prescribed Reata medicines. 

While yet to be approved, the press release reports that omaveloxolone has been granted Orphan Drug designation in Europe for the treatment of Friedreich’s ataxia and is currently under review by the European Medicine Agency.

Syfovre approved for geographic atrophy in advanced age-related macular degeneration

24th February 2023

According to a press release from the manufacturer Apellis Pharmaceuticals, syfovre (pegcetacoplan), is to become the first FDA approved therapy for geographic atrophy secondary to age-related macular degeneration which is a leading cause of blindness.

Geographic atrophy (GA) represents an advanced form of age-related macular degeneration and which leads to progressive and irreversible loss of vision. Age-related macular degeneration is a common condition with one US analysis suggesting an annual incidence of 3.5 per 1,000 individuals, around 293,000 new cases per year in those 50 years of age and older. Globally, the condition is estimated to affect some 5 million people and the median time to the development of central GA following any GA diagnosis is 2.5 years, with an average visual acuity decrease of 22 letters after 5 years.

The complement system is a component of the immune system that is central to the detection and destruction of invading pathogens and consists of three pathways. Moreover, dysregulation of this system is thought to be implicated in the development of age-related macular degeneration. Syfovre is a complement C3 inhibitor (i.e., one of these pathways) and data from a phase 2 trial showed that the drug produced significant reductions in the growth of GA compared to sham treatment.

Syfovre phase 3 clinical studies

The approval of syfovre was based on the data from two trials, OAKS and DERBY, both of which were randomised, sham trials of 24 months duration and where syfovre (15 mg/0.1ml) was administered either monthly or every other month. The results showed that in OAKS, monthly syfovre treatment reduced the rate of GA growth by 22% and by 18% when used every other month. Similarly, data from DERBY observed a 18 and 17% reduction in GA growth rate for monthly and alternate monthly injections respectively. Further details on the product can be found in the full prescribing information sheet.

According to the press release, syfovre is currently under review by the European Medicines Agency with a decision expected in early 2024.

FDA approves ruxolitinib cream for vitiligo

28th July 2022

Ruxolitinib cream has become the first FDA-approved treatment for the re-pigmentation of skin in patients with non-segmental vitiligo

According to a press release by the manufacturer Incyte, ruxolitinib cream has been approved by the US Food and Drug Administration for the treatment of non-segmental vitiligo.

Vitiligo is a common, autoimmune skin disorder, in which there is loss of melanocytes in the epidermis and presents clinically as well-demarcated, white patches on the body. It is estimated that the condition has a worldwide prevalence of 0.5%2%. Vitiligo has two main types, non-segmental and segmental with the former being symmetrical, whereas the latter is more localised. Non-segmental vitiligo is the more common form and accounts for 85–90% of cases. Ruxolitinib is a Janus kinase inhibitor and the cream has already showed anti-inflammatory and prompt antipruritic effects in the management of patients with atopic eczema.

The FDA decision to approve ruxolitinib (brand name Opzelura™) was based on the findings of two clinical trials, TRuE-V1 and TRuE-V2. Both were similar in design and evaluated the efficacy and safety of ruxolitinib cream in adolescent (12 years and older) and adult participants with non-segmental vitiligo for whom the vitiligo involved area (facial and non-facial) did not exceed 10% of their body surface area (BSA). In each of the trials, participants were randomised received ruxolitinib cream 1.5% or vehicle for 24 weeks which was applied twice daily. Following on from the initial 24 week period, participants were offered the opportunity to continue in the treatment extension arm for up to 52 weeks. The primary endpoint for both trials was the facial Vitiligo Area Scoring Index (F-VASI75) and a 75% improvement from baseline in the F-VASI (F-VASI75) has been identified as within-patient clinically meaningful thresholds.

Ruxolitinib cream: clinical efficacy

A dose ranging Phase II randomised trial did provide evidence for the effectiveness of the treatment. In the trial, 157 patients (mean age, 48·3 years, 46% male), with depigmentation of 0·5% or more of their facial body surface area (BSA) and 3% or more of their non-facial BSA, were randomised 1:1:1:1 to receive ruxolitinib cream (1·5% twice daily, 1·5% once daily, 0·5% once daily, or 0·15% once daily) or vehicle twice daily. The primary endpoint was a F-VASI50 after 24 weeks and this was achieved after 24 weeks by 45% of patients using the 1.5% cream twice daily and 50% of those using the same strength once daily compared to only 3% receiving placebo.

Although the results of TRuE-V1 and V2 are not yet published, the manufacturer’s press release reports that after 24 weeks, approximately 30% of patients using ruxolitinib cream achieved the primary endpoint compared with approximately 8% of those using the vehicle in TRuE-V1 and 13% in TRuE-V2. Additionally, after 24 weeks, more than 15% of those receiving ruxolitinib cream achieved a F-VASI90 (i.e., 90% improvement from baseline), compared to approximately 2% of patients treated with vehicle.  At week 52, approximately 50% of ruxolitinib cream patients achieved the primary endpoint and 30% had achieved a F-VASI90.

Although ruxolitinib cream has yet to be approved by the European Medicines Agency, it is currently under review by the organisation.