Thursday, August 31, 2017

New Treatment for SBMA Approved in Japan

Since the initial announcement in mid-August, there has been a lot of buzz about this treatment. Mike Wilson posted the following translation on the KD-Downunder Facebook page. The comments posted from others living with KD is interesting.

The article he is referring to can be found at  When this trial was first introduced back in 2010, I posted an article on it. You can read it here. This month I posted the announcement about the long-term trial results. It can be found here.  

Interesting news - A new treatment for SBMA just approved in Japan:

Leuplin SR® Injection Kit 11.25 mg in Japan

About supplemental approval of indication of "suppression of progression of spinal and bulbar muscular atrophy"

We are pleased to announce that "Spinal and Bulbar Muscular (Spinal and Bulbar Muscular Disease)" from the Ministry of Health, Labor and Welfare for "Leuprin® SR Injection Kit 11.25 mg" (generic name: leuprorelin acetate, We are pleased to announce that we have received additional indication of the suppression of the progression of "Atrophy: SBMA").

Leuplin SR is a 12-week sustained-release sustained-release preparation of highly active LH-RH agonist (luteinizing hormone-releasing hormone derivative) synthesized by Sumitomo Chemical, which acts continuously on the pituitary gland, It inhibits the production of sex hormones by reducing reactivity. This drug is used as a therapeutic agent for hormone dependent diseases such as prostate cancer and premenopausal breast cancer in Japan.

The acquisition of additional indication for the indication was mainly evaluated based on the results of doctor-initiated trials that examined the effectiveness and safety of Leuplin SR for patients with SBMA, centering on Nagoya University neurology department. It is world's first approval as a therapeutic agent for the progression of SBMA.

Toshiro Taniya, director of the Company's Japan Development Center, said, "To date, no effective treatment for SBMA has been established domestically and internationally, and drugs that could contribute to the treatment of this disease were sought. , It will become the world's first medicine to be useful in treating patients of SBMA.We appreciate the patients and doctors who cooperated in developing this drug, and for the patient and medical staff We will strive to deliver medicines for diseases with high unmet medical needs. "

About indications / effects, dosage and dosage approved this time

Indications and effects: inhibition of progression of spinal and bulbar muscular atrophy

Dosage / administration: Usually, adults receive 11.25 mg subcutaneously as Leuprorelin acetate once every 12 weeks.

Upon administration, push the plunger rod with the injection needle facing upward, move the whole amount of the suspension liquid to the powder part, and carefully suspend and use it while taking care not to foam.

About SBMA

SBMA is characterized by muscle atrophy and is an X-linked lower motor neuron disease that develops in adult male. Due to abnormal accumulation of mutant androgen receptor (AR) with polyglutamine in the nucleus, an androgen hormone dependent neuronal damage occurs. It usually develops around 30 to 60 years old, it follows a slow progressive course, not only forced to live in bed chair or bedridden life at the end of the year, but also repeats aspiration pneumonia. In Japan, it is stipulated as a designated intractable disease, and it is reported that the number of persons with specific medical care recipient 's passengers is 1,223 (Research on Specific Diseases by Ministry of Health, Labor and Welfare in FY2006).


  1. Is there any progress updates on Leuplin SR. my son is 33 and has been diagnosed. He suffers horrible cramps. My 1st son is 41 he too is diagnosed. It is a nightmare to watch them suffer with cramps that seem to last all night

  2. Thank you for your inquiry. It is difficult to watch a loved one in pain and not be able to help.

    Your sons need to discuss potential treatments for KD with their neurologists. Leuprorelin has been studied for several years in Japan. There appears to be some benefit (slowing) of the progression, but the side effects need to be carefully considered before moving forward. Dutasteride is another option that appears to help some with KD. Again, there are side effects. Currently, there is no known cure for KD.

    Ref. cramping, there are treatments to reduce the severity of the cramping. Again, this topic needs to be discussed with the neurologist.

    Your sons are young and there is a possibility a treatment or cure will be discovered within their lifetime. Keep the faith.


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