Sickle Cell Disease (SCD) is a rare blood disorder that affects the hemoglobin involved in delivers oxygen to cells throughout the body. It is inherited in an autosomal recessive trait in which both copies of the genes present in each cell have mutations. The parents of an individual with an autosomal recessive condition comprised of one copy of the mutated gene, and do not show signs and symptoms of the condition. The atypical hemoglobin molecules called hemoglobin S are present among the patients. Sickle cell disease is characterized by the presence of sickle or crescent-shaped, red blood cells in the bloodstream. Sickle Cell Disease occurred due to the mutations in the hemoglobin beta (HBB) gene.
Sickle Cell Disease Epidemiology Insights
The United States accounts for the highest prevalent cases of Sickle cell disease followed by the EU5 countries (France, Germany, Spain, Italy, and the United States), and Japan
According to the Centers for Disease Control and Prevention (CDC), Sickle Cell Disease affects approximately 100,000 Americans
Sickle Cell Disease occurs among about 1 out of every 365 Black or African-American births
Sickle Cell Disease occurs among about 1 out of every 16,300 Hispanic-American births
Approximately, 1 in 13 Black or African-American babies is born with sickle cell trait (SCT).
The mutations in the HBB gene are common in people from African, Mediterranean, Middle Eastern, and Indian ancestry and in people from the Caribbean and parts of Central and South America, but can be found in people of any ethnicity
Every year June 25 is celebrated as the World Vitiligo Day to raise awareness regarding Vitiligo, fight prejudice, and raise funds for research, support & education. The aim of this day is to include the recognition of the bullying, social neglect, psychological trauma, and disability of millions of people affected by vitiligo. The primary purpose of this day is to raise money for research, give free skin exams, and educate physicians on how to best take care of patients with vitiligo.
Vitiligo is the long term skin condition in which the skin loses the pigment i.e. melanin, essential for determining the color of skin, hair, and eyes. This leads to the slow growth of the white patches of irregular shapes on the skin. Vitiligo can also affect the mucous membranes, including tissues inside the mouth and nose. It can affect people of all skin types but is generally noticeable much in darker skin people.
Around 1 out of 10 people i.e. 1-2% of people are suffering from Vitiligo worldwide
The United States accounts for the highest prevalent cases of Vitiligo in comparison to the EU5 (Germany, France, Spain, Italy, and the United Kingdom) and Japan
Vitiligo is classified as the non-segmental, and segmental Vitiligo. Non-segmental Vitiligo is most prevalent in comparison to the segmental Vitiligo
Approximately, 95% of the people develop the condition prior to age 40.
Around 20% of people have a family member suffering from Vitiligo
Males and females are almost equally affected by Vitiligo
Vitiligo is sometimes associated with certain other medical conditions, including thyroid dysfunction, diabetes mellitus, Addison’s disease, etc.
Several factors such as autoimmune disease, genetic factors, sunburn or any cut, oxidative stress, neurochemicals, and exposure to the industrial chemicals increase the risk of developing Vitiligo
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
CD30+ Lymphproliferative Disorders Primary cutaneous anaplastic large cell lymphomaLymphomatoid papulosis
Extranodal natural killer/T-cell lymphoma, nasal type
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.
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.
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.