Monday, 19 October 2015

Spinal muscular atrophy (SMA) is a hereditary autosomal recessive disease affecting areas of the nervous system that control voluntary muscle movement. SMA is identified as a leading genetic disorder causing mortality among infants. According to Orphanet, one infant in 6,000 births is estimated to suffer from SMA. Furthermore, over 10 million people in the U.S. are carriers of SMA gene. Depending on the age of onset and symptoms, spinal muscular atrophy is classified in four main types. Type 1 SMA is the most severe form, and affects infants aged between zero and six months. Over 60% of annual SMA cases are of type 1 SMA, wherein infants usually do not survive for more than 2 years. Type 2 SMA is a moderate form affecting children aged between six months and eighteen months. Patients with Type 3 SMA and Type 4 SMA usually have a normal lifespan.
Currently, no cure exists for spinal muscular atrophy, and treatment is directed towards managing common symptoms of SMA. Physiotherapy is strongly recommended in early stages of the disease to extend range of motion and improve muscle flexibility. Other treatment methods are targeted to treat gastrointestinal and respiratory disorders, which are common in SMA patients. The SMA pipeline is dry, with single drug in phase 3, three in phase 2, and one in phase 3 clinical trials. ISIS-SMNRx by Isis Pharmaceutical is an antisense drug that has shown promising results in phase 2 clinical trials. Phase 3 study of ISIS-SMNRx for treatment of Type 1 SMA and Type 2 SMA is expected to complete in 2016. In the United States, the drug is expected to reach annual sales of USD 0.47 billion by 2023. Olesoxime (TRO19622) has completed its phase2/3 trials, and has shown promising results, with potential to effectively treat type 2 and type 3 SMA. Other potential candidates include Olesoxime (TRO19622), LMI070, RO6885247, CK-2127107, scAAV9.CB.SMN and RG3039.

The United States and Europe have higher percentage of diagnosed cases of SMA. Government and private organization in these regions are also involved in creating awareness about the disease. Higher confirmed carrier population would also help in faster market growth in these regions. Furthermore, government incentives for development of therapeutic alternatives for rare diseases also boosts research and development in the SMA landscape.Companies with potential candidates in the spinal muscular atrophy pipeline include AveXis, Inc., Cytokinetics, Inc., F. Hoffmann-La Roche Ltd., Novartis AG, and Isis Pharmaceuticals, Inc.   

Spinal Muscular Atrophy Pipeline is segmented as below:
Late Stage Candidates (Phase 3)

Early Stage Candidates (Phase 1 and Phase 2)

Olesoxime (TRO19622)

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Wednesday, 14 October 2015

Angina is a kind of chest pain or discomfort caused by poor flow of blood in the heart. This often occurs due to narrowing of blood arteries (atherosclerosis) caused by plaque formation or blood clot. People with obesity, history of heart disease, diabetes, high level of blood cholesterol and smoking are high risk of getting affected with angina. Some common symptoms associated with angina include pain, pressure or strange feeling in chest, shortness of breath, lightheadedness, irregular heartbeat, nausea or vomiting and weakness. Angina may be of two types: stable angina and unstable angina. Stable angina is the most common type of angina and is characterized by predictable pattern of chest pain. On the other hand, unstable angina has no typical pattern of chest pain. Stable angina happens when the heart does not get enough oxygen to perform a work which has been asked to do such as exercise or lifting heavy weight which may also occur due to cold weather and emotional stress.

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Diagnosis of chronic stable angina includes medical history examination by a doctor and few tests such as coronary angiography, coronary risk profile (special blood tests), ECG, exercise tolerance test (stress test or treadmill test), stress echocardiogram and nuclear medicine (thallium) stress test. The treatment options for chronic stable angina include lifestyle changes such as daily exercise and quitting smoking, medications, and procedures such as angioplasty and stent placement and sometimes heart bypass surgery. Most commonly used medications include nitrates, ACE inhibitors and calcium channel blockers. Nicorandil, ranolazine and ivabradine are some of the latest anti-anginal agents which are also used for treating chronic stable angina.

The global market for chronic stable angina therapeutics is expected to grow at a moderate CAGR. Some major factors driving the market growth include high prevalence of the disease, extensive research and development of new more effective molecules. According to National Institute for Health and Clinical Excellence, an estimated 2 million people suffered from with stable angina in England in 2010. American Heart Association claims that around 9.1 million people are living with stable angina in the United States. Since stable angina cannot be cured immediately, it is expected that the base population of affected people will increase over the years and such high prevalence will drive market growth particularly in developed countries. The market for chronic stable angina therapeutics is likely to face significant challenges owing to patent expiry of major drugs such as bivalirudin (Angiomax/Angiox) in 2015 in Europe and rosuvastatin (Crestor) in 2016. These patent expiries are expected to attract generic manufacturers who will intensify the competition by selling drugs at low prices.

Geographically, North America represents the largest market, followed by Europe, Asia-Pacific and Rest of the World. In Asia-Pacific, rapid urbanization leading to life style changes and emerging economy of key countries such as India and China are expected to drive the market growth. Obesity, stress and cardiovascular diseases have become very common and will drive the market in developing countries significantly. Rapidly evolving medical tourism industry will also play a key role in driving the market growth in the region as a large number of people come from foreign countries to countries like India, Indonesia, Thailand and Singapore for the treatment of chronic stable angina.

There are many pharmaceutical companies and research institutes which are engaged in the development, manufacturing and marketing of drugs for treating chronic stable angina. Some major companies and research institutes include Pfizer, Inc., Mylan, Inc., Actavis Inc., Chong Kun Dang Pharmaceutical Corp., Roxane Laboratories, Inc., Gilead Sciences, Inc., CNS, Inc., Nanjing University and King's College London.

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Geographies analyzed under this research report include
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Asia Pacific
Rest of the World 

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