STOCKHOLM, April 25, 2024 /PRNewswire/ -- Medivir AB(NASDAQ: MVIR) (STO: MVIR), a pharmaceutical company focused on developing innovative treatments for cancer in areas of high unmet medical need, announced today that its selective cathepsin K inhibitor, MIV-711, has been granted Rare Pediatric Disease Designation (RPDD) as well as Orphan Drug Designation (ODD) for the treatment of Legg-Calvé-Perthes Disease (LCPD)..

斯德哥尔摩，2024年4月25日/PRNewswire/--Medivir AB（纳斯达克：MVIR）（STO：MVIR）是一家专注于在医疗需求未得到满足的地区开发癌症创新治疗方法的制药公司，今天宣布其选择性组织蛋白酶K抑制剂MIV-711已被授予罕见儿科疾病（RPDD）和孤儿药物（ODD），用于治疗Legg Calvé-Perthes病（LCPD）。。

The FDA defines a rare pediatric disease as a serious or life-threatening disease in which the disease manifestations primarily affect individuals aged from birth to 18 years of age. Pediatric diseases recognized as 'rare', affect fewer than 200,000 people in the United States. With a RPDD, MIV-711 qualifies to receive fast track review and upon marketing approval in LCPD, the Company may be eligible to receive a Priority Review Voucher from the FDA.

FDA将罕见的儿科疾病定义为严重或危及生命的疾病，其中疾病表现主要影响从出生到18岁的个体。被认为是“罕见”的儿科疾病，在美国影响不到20万人。通过RPDD，MIV-711有资格接受快速审查，并且在LCPD获得营销批准后，该公司可能有资格获得FDA的优先审查凭证。

The voucher can be redeemed to receive priority review for any subsequent marketing application or may be sold to another company for their use..

凭单可以兑换以获得后续任何营销应用程序的优先审查，也可以出售给其他公司供其使用。。

- 'LCPD is a disease, with a significant impact on daily life as well as risk for long-term sequalae, for which there are no available effective treatments. Children with LCPD are more likely to suffer from obesity and depression due to the forced immobility as a consequence of the disease. In more than half of the children, the affected leg becomes shorter than the healthy leg and about 50% end up with a deformed hip joint where ultimately osteoarthritis develops.

-“LCPD是一种疾病，对日常生活产生重大影响，并有长期感染红杉的风险，目前尚无有效的治疗方法。患有LCPD的儿童由于疾病而被迫不动，更容易患肥胖症和抑郁症。在超过一半的儿童中，受影响的腿比健康的腿短，大约50%的儿童最终会出现髋关节变形，最终会发展为骨关节炎。

We are delighted that MIV-711 has been granted RPDD by the FDA with the potential to become the first approved treatment option. We see partnership as an attractive way to move forward with MIV-711 to ensure speed of development,' said Jens Lindberg, CEO of Medivir..

我们很高兴MIV-711已被FDA授予RPDD，有可能成为第一个批准的治疗选择。Medivir首席执行官延斯·林德伯格（JensLindberg）说，我们认为合作伙伴关系是推动MIV-711发展以确保发展速度的一种有吸引力的方式。。

To gain RPDD, there must be supportive data suggesting that the drug may be effective in the disease. MIV-711 has shown, in an LCPD-specific animal model, the ability to prevent femoral head deformity and positive impact on biomarker of bone degradation without negatively impacting normal bone formation.

为了获得RPDD，必须有支持性数据表明该药物可能对该疾病有效。MIV-711在LCPD特异性动物模型中显示出预防股骨头畸形的能力，并对骨降解的生物标志物产生积极影响，而不会对正常骨形成产生负面影响。

Similarly, clinical studies have shown that MIV-711 prevents bone degradation in patients with osteoarthritis, further supporting the potential clinical benefit in LCDP..

同样，临床研究表明，MIV-711可防止骨关节炎患者的骨降解，进一步支持LCDP的潜在临床益处。。

Cathepsin K is the main cysteine protease involved in bone resorption and cartilage degradation by osteoclasts, through the breakdown of key bone matrix proteins. Cathepsin K inhibition protects the damaged bone by reducing bone resorption and promoting bone formation, thereby addressing the key mechanisms causing pathological changes in LCPD, with potential to minimize long-term negative effects.

组织蛋白酶K是主要的半胱氨酸蛋白酶，通过关键骨基质蛋白的分解，参与破骨细胞的骨吸收和软骨降解。组织蛋白酶K抑制通过减少骨吸收和促进骨形成来保护受损的骨，从而解决导致LCPD病理变化的关键机制，并有可能将长期负面影响降至最低。

Selective inhibition of cathepsin K has the potential to provide clinical benefit in diseases characterized by excessive bone resorption, including additional pediatric bone disorders..

组织蛋白酶K的选择性抑制有可能为以过度骨吸收为特征的疾病（包括其他小儿骨骼疾病）提供临床益处。。

For additional information, please contact:

有关更多信息，请联系：

Magnus Christensen, CFO, Medivir AB

Magnus Christensen，Medivir AB首席财务官

Telephone: +46 8 5468 3100.

电话：+46 8 5468 3100。

E-mail: magnus.christensen@medivir.com

电子邮件：magnus.christensen@medivir.com

About Legg-Calvé-Perthes Disease

关于Legg-Calvé-Perthes病

Legg-Calvé-Perthes disease (LCPD) is a childhood hip disorder initiated by a disruption of blood flow to the head of the femur. Due to the lack of blood flow, the bone dies (osteonecrosis or avascular necrosis) and stops growing. Over time, healing occurs by new blood vessels infiltrating the dead bone and removing the necrotic bone which leads to a loss of bone mass and a weakening of the femoral head.

Legg-Calvé-Perthes病（LCPD）是一种儿童髋关节疾病，由股骨头血流中断引起。由于缺乏血流，骨骼死亡（骨坏死或缺血性坏死）并停止生长。随着时间的推移，愈合是通过新血管浸润死骨并去除坏死骨而发生的，这导致骨量减少和股骨头减弱。

The condition is most commonly found in children between the ages of 4 and 8, but it can occur in children between the ages of 2 and 15. It is estimated to affect about 10 in 100.000 children per year in the USA. It results in permanent deformity of the femoral head in many children, which increases the risk of developing osteoarthritis in adults..

这种情况最常见于4至8岁的儿童，但也可能发生在2至15岁的儿童身上。据估计，在美国，每年每10万名儿童中就有10名受到影响。它会导致许多儿童的股骨头永久畸形，从而增加成人患骨关节炎的风险。。

About MIV-711

关于MIV-711

MIV-711 is a potent and selective inhibitor of cathepsin K, the principal protease involved in breaking down collagen in bone and cartilage. It has been shown to slow, stop or reverse the progressive degeneration of joints affected by osteoarthritis. By inhibiting cathepsin K and increased/excessive activity of osteoclasts, MIV-711 has the potential to prevent degradation of bone and cartilage and counteract the osteonecrosis induced by lack of blood flow, to prevent femoral head deformity and long-term negative effects as well as positively impact Quality of Life..

MIV-711是组织蛋白酶K的有效和选择性抑制剂，组织蛋白酶K是分解骨骼和软骨中胶原蛋白的主要蛋白酶。它已被证明可以减缓，停止或逆转受骨关节炎影响的关节进行性退化。通过抑制组织蛋白酶K和破骨细胞活性的增加/过度，MIV-711有可能防止骨和软骨的降解，抵消由于缺乏血流引起的骨坏死，防止股骨头畸形和长期负面影响以及积极影响生活质量。。

About Medivir

关于Medivir

Medivir develops innovative drugs with a focus on cancer where the unmet medical needs are high. The drug candidates are directed toward indication areas where available therapies are limited or missing and there are great opportunities to offer significant improvements to patients. Medivir is focusing on the development of fostroxacitabine bralpamide (fostrox), a smart, targeted chemotherapy designed to selectively treat liver cancer cells and to minimize side effects.

Medivir开发创新药物，重点关注未满足医疗需求的癌症。候选药物针对可用疗法有限或缺失的适应症领域，并且有很大的机会为患者提供显着改善。Medivir专注于开发fostroxacitabine bralpamide（fostrox），这是一种智能的靶向化疗，旨在选择性治疗肝癌细胞并尽量减少副作用。

Collaborations and partnerships are important parts of Medivir's business model, and the drug development is conducted either by Medivir or in partnership. Medivir's share (ticker: MVIR) is listed on Nasdaq Stockholm's Small Cap list. www.medivir.com..

合作和伙伴关系是Medivir商业模式的重要组成部分，药物开发由Medivir或合伙进行。Medivir的股票（股票代码：MVIR）在纳斯达克斯德哥尔摩的小盘股名单上上市。www.medivir.com。。

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以下文件可供下载：

https://mb.cision.com/Main/652/3967577/2759269.pdf

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Press release (PDF)

新闻稿（PDF）

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SOURCE Medivir

来源Medivir

Company Codes: Bloomberg:MVIR@SS, ISIN:SE0020181014, RICS:MVIR.ST, Stockholm:MVIR

公司代码：彭博社：MVIR@SS，ISIN:SE0020181014，RICS:MVIR。斯德哥尔摩ST：MVIR