Notice of Orphan Drug Designation for JR-446 for Mucopolysaccharidosis Type IIIB by the U.S. FDA
TOKYO & HYOGO, Japan--(BUSINESS WIRE)-- MEDIPAL HOLDINGS CORPORATION (TSE 7459, MEDIPAL) and JCR Pharmaceuticals Co., Ltd. (TSE 4552, JCR) today announced that the U.S. Food and Drug Administration (FDA) granted orphan drug designation (ODD) to JR-446, an investigational drug for the treatment of mucopolysaccharidosis type IIIB (MPS IIIB or Sanfilippo syndrome type B).
MPS IIIB affects an estimated 500 to 1,000 individuals worldwide, 1 causing severe central nervous system (CNS) symptoms. Despite the dire need, there are currently no approved treatments available for this condition. JR-446, developed using JCR's proprietary J-Brain Cargo ® technology, has shown promising non-clinical results in addressing the CNS symptoms of this challenging disorder, and is currently being studied in a Phase I/II trial that is being conducted in Japan (JR- 446-101) under a collaboration agreement between the two companies.
'We are extremely pleased that JR-446 has received orphan drug designation from the U.S. FDA,' said Shuichi Watanabe, Representative Director, President and CEO of MEDIPAL. 'We will continue the development of JR-446 in collaboration with JCR so that we can soon deliver this drug to the patients and their families who are desperately waiting, as there is currently no treatment available.'
'This orphan drug designation is an important development step for the JR-446 clinical program,' said Shin Ashida, President, Chairman and CEO of JCR Pharmaceuticals. 'This orphan drug designation supports the potential of this therapy to address the significant unmet medical need of patients with MPS IIIB.'
In September 2023, MEDIPAL and JCR entered into a licensing agreement in which MEDIPAL will commercialize JR-446 outside of Japan. In addition, MEDIPAL will support JCR in the clinical development of JR-446 in Japan, including the distribution of investigational drugs, disease awareness, and clinical trial advancement. 2
With the ODD, JR-446 will be eligible for various incentives to encourage the development in the U.S.
About Orphan Drug Designation in the U.S.
The U.S. FDA's Office of Orphan Products Development grants orphan status to drugs being developed to treat, prevent, or diagnose a rare disease or condition affecting fewer than 200,000 people in the U.S. The designation provides significant incentives to promote the development of the drug including the potential for market exclusivity for seven years upon FDA approval, eligibility for tax credits for qualified clinical trials, and waiver of Prescription Drug User Fee Act Application fee.
About Mucopolysaccharidosis Type IIIB (Sanfilippo Syndrome Type B) Mucopolysaccharidosis type IIIB, or Sanfilippo syndrome type B, is an autosomal recessive disease caused by pathogenic mutations in the NAGLU gene, encoding a lysosomal enzyme involved in the degradation of heparan sulfate. With the accumulation of heparan sulfate in the central nervous system in the brain, individuals with this condition present rapid neurological decline, including sleep disorders, loss of speech, and behavioral changes, which may significantly affect the quality of life of patients and their families.
About the J-Brain Cargo ® Platform Technology
JCR Pharmaceuticals has developed a proprietary blood-brain barrier-penetrating technology, J- Brain Cargo ®, to bring biotherapeutics into the central nervous system. The first drug developed based on this technology is IZCARGO ® (INN: pabinafusp alfa) and was approved in Japan for the treatment of a lysosomal storage disorder.
About MEDIPAL HOLDINGS CORPORATION
MEDIPAL is a holding company which controls, administers and supports the operating activities of companies in which it holds shares in the Prescription Pharmaceutical Wholesale Business; the Cosmetics, Daily Necessities and OTC Pharmaceutical Wholesale Business; and the Animal Health Products and Food Processing Raw Materials Wholesale and Related Business, and conducts business development for the MEDIPAL Group. For more information, visit https://www.medipal.co.jp/english/.
About JCR Pharmaceuticals Co., Ltd.
JCR is a global specialty pharmaceuticals company dedicated to advancing treatments for rare and genetic diseases. With nearly 50 years of expertise in Japan, JCR is expanding to the US, Europe, and Latin America. JCR's innovative therapies address conditions like growth disorder, MPS II, Fabry disease, acute graft-versus-host disease, and renal anemia. JCR is also developing treatments for rare diseases like MPS I, MPS II, MPS IIIA and B, and more. The core values of reliability, confidence, and persistence drive JCR's mission to enhance global medical progress. For more information, visit https://www.jcrpharm.co.jp/en/site/en/.
Cautionary Statement Regarding Forward-Looking Statements
This document contains forward-looking statements that are subject to known and unknown risks and uncertainties, many of which are outside our control. Forward-looking statements often contain words such as 'believe,' 'estimate,' 'anticipate,' 'intend,' 'plan,' 'will,' 'would,' 'target' and similar references to future periods. All forward-looking statements regarding our plans, outlook, strategy and future business, financial performance and financial condition are based on judgments derived from the information available to us at this time. Factors or events that could cause our actual results to be materially different from those expressed in our forward-looking statements include, but are not limited to, a deterioration of economic conditions, a change in the legal or governmental system, a delay in launching a new product, impact on competitors' pricing and product strategies, a decline in marketing capabilities relating to our products, manufacturing difficulties or delays, an infringement of our intellectual property rights, an adverse court decision in a significant lawsuit and regulatory actions. This document involves information on pharmaceutical products (including those under development). However, it is not intended for advertising or providing medical advice. Furthermore, it is intended to provide information on our company and businesses and not to solicit investment in securities we issue. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the factors that could cause actual results to differ materially, even if new information becomes available in the future.
References
Based on data from JCR's own investigations, referring to the Ministry of Health, Labour and Welfare's public research.
Reference: Press release on the licensing agreement for JR-446 between MEDIPAL and JCR (September 28, 2023).
Hashtags
Try Our AI Features
Explore what Daily8 AI can do for you:
Comments
No comments yet...
Related Articles
Yahoo
12 minutes ago
- Yahoo
ProZenith Launches Natural Supplement Formulated for Weight Management Support
U.S.-Made Product Supports General Wellness Goals with a 60-Day Refund Policy Aurora, June 06, 2025 (GLOBE NEWSWIRE) -- ProZenith recently announced the launch of its new wellness supplement developed to assist individuals in maintaining energy, focus, and mindful appetite awareness as part of a balanced and active lifestyle. Manufactured in the United States in a facility that is FDA-registered and GMP-certified, the product is now available through official online channels. ProZenith is intended for individuals pursuing support for general weight management and overall well-being. Its formulation includes select ingredients chosen to align with healthy routines and support individuals seeking help managing non-hunger-related snacking behaviors. Each purchase of ProZenith is covered by a 60-day refund policy, reflecting the company's customer-first return assurance framework. All ProZenith supplements are manufactured without genetically modified ingredients and adhere to U.S. quality standards. Production takes place in an FDA-registered facility that complies with current Good Manufacturing Practices (cGMP). ProZenith is currently available through the company's official online platform at with multiple purchase options provided for convenience. About ProZenith ProZenith develops nutritional supplements designed to support individuals on their wellness journeys. The company emphasizes high-quality manufacturing, transparency, and customer satisfaction as it continues to expand its product offerings for health-conscious consumers. For customer support inquiries, contact:support@ Disclaimer:This product has not been evaluated by the Food and Drug Administration. This product is not intended to diagnose, treat, cure, or prevent any disease. Media Contact: Company: ProZenith Address: 19655 E 35th Dr #100, Aurora, CO 80011 Email: support@ Order Phone Support: (925) 217-7353 CONTACT: Company: ProZenith Address: 19655 E 35th Dr #100, Aurora, CO 80011 Email: support@ Order Phone Support: (925) 217-7353Sign in to access your portfolio
Yahoo
14 minutes ago
- Yahoo
Earn up to 110,000 bonus miles with a new Delta SkyMiles credit card
American Express and Delta are out with all-new welcome bonus offers across three personal credit cards within the Delta SkyMiles suite: Delta SkyMiles® Gold American Express Card: Earn 80,000 bonus miles after spending $3,000 on eligible purchases in your first six months of card membership. Delta SkyMiles® Platinum American Express Card: Earn 90,000 bonus miles after spending $4,000 on eligible purchases in your first six months of card membership. Delta SkyMiles® Reserve American Express Card: Earn 100,000 bonus miles after spending $6,000 on eligible purchases in your first six months of card membership. There are also new offers on Delta business cards: Delta SkyMiles® Gold Business American Express Card: Earn 90,000 bonus miles after spending $6,000 on eligible purchases in your first six months of card membership. Delta SkyMiles® Platinum Business American Express Card: Earn 100,000 bonus miles after spending $8,000 on eligible purchases in your first six months of card membership. Delta SkyMiles® Reserve Business American Express Card: Earn 110,000 bonus miles after spending $12,000 on eligible purchases in your first six months of card membership. With over 290 destinations across six continents and more than 200 million customers served in 2024, Delta is one of the most well-known airlines worldwide. And you could reduce much of the upfront cost of flying to one of Delta's global destinations if you're able to take advantage of these generous sign-up bonuses. It depends on how you redeem the miles, but we value Delta SkyMiles at an average of 1.18 cents each. That means 100,000 miles equals about $1,180. However, as with many airline credit cards, the exact value of your miles depends on how you use them. Since Delta uses dynamic pricing, you can see different values with each redemption. You can find plenty of value with 100,000 (give or take, depending on the welcome offer) Delta SkyMiles. This is especially true when accounting for the TakeOff 15 benefit, which provides an automatic 15% discount on eligible award flights for Delta SkyMiles cardholders. Let's take a look at some possible redemptions. You can fly from Los Angeles (LAX) to Auckland (AKL) for 38,200 miles. The cash price for the same flight is $1,341, giving you a redemption value of about 3.5 cents per mile ($1,341 / 38,200 = 0.035104). You'll still have plenty of miles left to figure out your return flight, which could mean including a stop in Australia after visiting Hobbiton (and while you're in the area). This flight from Salt Lake City (SLC) to Mexico City (MEX) will only set you back 26,300 miles. The redemption value for this flight is about 1.5 cents per mile, which is better than our average valuation. And with miles to spare, you can bring some friends or work on covering the return leg home. You don't have to get fancy with your redemptions; a short flight will do just fine if it saves you money, especially if it's a route you frequently take. Consider this example: A flight between Atlanta (ATL) and Miami (MIA) that costs 8,300 miles or $117. With 100,000 miles, you could take this flight 12 times, giving you plenty of opportunities for vacations or visiting friends and family. This particular flight has a redemption value of about 1.4 cents per mile. See which flavor — Gold, Platinum, or Reserve — of Delta SkyMiles card is the best fit for you. Consider which Delta SkyMiles business card is right for you if you run a small best Delta SkyMiles credit card for you depends on your goals and spending habits. If you only fly occasionally, maybe a few times per year, we recommend the Delta SkyMiles Gold Amex Card. This card has the lowest annual fee but still provides one of the best benefits: a free checked bag. With only a few roundtrip flights, you can offset the annual fee in saved baggage charges. If you travel a lot, a card like the Delta SkyMiles Platinum Amex Card or Delta SkyMiles Reserve Amex Card may make more sense. These cards have higher annual fees, but they also provide more benefits. For example, the Delta Reserve Amex Card offers Delta Sky Club Access (at least 15 visits each Medallion year), which could come in handy the more you find yourself in airports. Business owners can apply for the personal or business versions of the Delta SkyMiles credit cards. The business credit cards are largely the same as their personal variants, but their welcome offers require increased spending, so keep that in mind when making your decision. All the Delta SkyMiles credit cards associated with these new welcome offers have annual fees. If you want to avoid an annual fee at all costs, consider no-annual-fee credit Disclosure: The information in this article has not been reviewed or approved by any advertiser. All opinions belong solely to the Yahoo Finance and are not those of any other entity. The details on financial products, including card rates and fees, are accurate as of the publish date. All products or services are presented without warranty. Check the bank's website for the most current information. This site doesn't include all currently available offers. Credit score alone does not guarantee or imply approval for any financial product.
Yahoo
an hour ago
- Yahoo
Tampa Police get 300 new lifesaving choking devices from New York-based company
The Brief A company based in New York donated choking rescue devices to the Tampa Police Department. The devices, which are called LifeVac, create a one-way suction to remove lodged food or objects from a choking victim. These devices will be distributed to officers soon. TAMPA, Fla. - A New York-based company donated 300 choking rescue devices to the Tampa Police Department. These devices, called LifeVac, create a one-way suction to remove lodged food or objects from a choking victim. "This is just another tool for us, especially for our youth, to be out there and provide another opportunity to save a life," Tampa Police Chief Lee Bercaw said of the donation. The backstory Last month,a Tampa police officer used back blows to help save a toddler who was choking on a tomato. "That's the reason that we're getting this donation," Bercaw said Friday of the bodycam video. "[LifeVac inventor] Mr. Lih saw that video and said, 'I've got to get that product down here in a generous donation.'" "I knew as a parent, I'd be freaking out. I would be very distressed. So I tried to come up with something really simple. You push it, pull it, and that suction pulls it out," LifeVac President and Creator Arthur Lih said. These devices will be distributed to officers soon. Bercaw told FOX 13 the devices will be placed in patrol cars and will be used in schools and at Police Athletic League youth activities. Dig deeper As shown in LifeVac's online instructional video, the New York-based company said people should first follow choking protocols established by the American Red Cross. That means doing back blows and abdominal thrusts first. And if those actions don't work – or if those actions cannot be completed for whatever reason – then a LifeVac device can come into play. The U.S. Food and Drug Administration (FDA) encourages people to follow established choking rescue protocols by the American Red Cross and the American Heart Association. In an April 2024 update, the FDA said these protocols do not include anti-choking devices: "The safety and effectiveness of anti-choking devices that are being sold over-the-counter have not been established; they are not FDA approved or cleared." Success Stories Just this week, a Georgia police officer used a LifeVac device to save a driver choking on a water bottle cap. Years ago, LifeVac devices were placed in every school in Sarasota County. The Source The information in this story was gathered by FOX 13's Ariel Plasencia.
