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Treatments for rare skin diseases receive high-level approvals

Article-Treatments for rare skin diseases receive high-level approvals

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Skin diseases do not only impact physical appearance but also leave a lasting psychological impact on patients. However, many diseases, even the rare ones that are typically not curable, respond well to treatment.

According to Cleveland Clinic, skin diseases are conditions that affect the skin and may cause rashes, inflammation, itchiness, or other skin changes. Some skin conditions may be genetic, while lifestyle factors may cause others. Skin disease treatment may include medications, creams or ointments, or lifestyle changes.

Lesser-known dermatology diseases

Experts say that there is a wide range of dermatology-related diseases, and many are not widely known by the general population in UAE and worldwide, such as lichen planus, pemphigus vulgaris, erythema, vitiligo, and multiforme, to name a few.

Dr. Reem Al Kaddah, Specialist Dermatologist, Medcare Medical Centre.jpg

Dr. Reem Al Kaddah, Specialist Dermatologist, Medcare Medical Centre, Motor City.

“The impact of these conditions can range from mild such as lichen planus to more serious conditions like pemphigus vulgaris that can adversely affect life expectancy,” said Dr. Reem Al Kaddah, Specialist Dermatologist, Medcare Medical Centre, Motor City.

“Lichen planus can affect skin, hair, nails, or even oral mucosa. For the most part, it causes the skin to be itchy and develops raised papules and rashes that can affect any part of the body, but especially limbs. Pemphigus vulgaris will affect ages 30 to 60 years old. The most common symptoms include painful blisters, erosions affecting the skin and mucosal surfaces in the mouth,” she said.

Likewise, vitiligo is a pigmentation disorder of the skin and mucous membranes that affects 0.5-2 per cent of the world population over 20 years old. “It is not contagious or life-threatening, but it does last for life which patients can find upsetting and depressing,” explained Dr. Laith Alshar, Specialist Dermatologist, Clinical and Aesthetics at Fakeeh University Hospital, Dubai.

Dr. Laith Alshar, Specialist Dermatologist, Clinical and Aesthetics at Fakeeh University Hospital.jpg

Dr. Laith Alshar, Specialist Dermatologist, Clinical and Aesthetics at Fakeeh University Hospital, Dubai.

Nonsegmental vitiligo is the most common form of vitiligo. The condition involves loss of pigment (depigmentation) in patches of skin. Depigmentation may occur on the face, neck, and scalp, and around body openings such as the mouth and genitals, as well as areas that tend to experience rubbing or impact, such as the hands and arms.

Spesolimab and Opzelura receive FDA approvals

Recently, the US Food and Drug Authority (FDA) became the first regulatory authority to approve spesolimab as a treatment option for generalised pustular psoriasis (GPP) flares in adults. GPP is a rare disease, and its impact on those affected is immense. Patients experience widespread non-infectious pustules erupting on the skin, which are red, itchy, painful, and visible to others.

To help combat the disease, Boehringer Ingelheim launched spesolimab, a novel, selective antibody that blocks the activation of the interleukin-36 receptor (IL-36R), a signaling pathway within the immune system shown to be involved in the pathogenesis of GPP.

Dr. Mohamed Meshref, Medical Director for India, Middle East, Turkey and Africa (IMETA) region, Boehringer Ingelheim, said: “GPP is a dermatological condition that can have a profound impact on a patient's quality of life. Flares associated with GPP are common in people living with this devastating skin condition and if left untreated, they can be life-threatening. This approval by the US FDA is a turning point and will help make a difference for patients living with the rare condition.”

Spesolimab has received Breakthrough Therapy Designation in the US, China, and Taiwan, Priority Review in the US and China, Orphan Drug Designation in the US, Korea, Switzerland, and Australia, Rare Disease Designation, and fast track in Taiwan, for the treatment of GPP flares.

Treatment options for diseases such as lichen planus range from topical steroids to systemic oral medications such as acitretin methotrexate azathioprine. “The challenge with these medications is the ability to monitor compliance given the side effects and the need to regularly monitor lab tests. The same also applies to pemphigus vulgaris medications with additional biologic treatments such as rituximab which has FDA approval,” said Dr. Al Kaddah.

For vitiligo, the FDA has approved Opzelura (ruxolitinib) cream for the treatment of non-segmental vitiligo in adult and pediatric patients 12 years of age and older.

According to Dr. Alshar, the understanding of the pathogenesis of vitiligo develops in parallel with the knowledge of the genetic regulation of the phenomena linked to oxidative stress and the cutaneous immune response.

“The epigenetic control of the gene expression linked to these phenomena can be an integrator of these processes since environmental and dietary factors, behavioural response patterns to stress, and the increasing exposure to industrialised compounds are intensely present in modern life, especially air pollution and aromatic compounds,” he said.

Three emerging vitiligo therapeutic classes are at the most advanced stage of development and are associated with the most important pathogenic pathways which are the tyrosine kinase inhibitors.

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