Santhera's AGAMREE Receives NMPA Approval for DMD Treatment

Santhera Achieves Milestone with AGAMREE Approval in China

Santhera Pharmaceuticals is proud to announce that its drug, AGAMREE® (vamorolone), has received the green light from China’s National Medical Products Administration (NMPA) for the treatment of Duchenne muscular dystrophy (DMD) in children aged four years and older. This significant approval marks AGAMREE as the first and only authorized therapy for DMD in the country, representing a breakthrough for many families affected by this heartbreaking condition.

The Importance of AGAMREE for DMD Treatment

DMD is a rare, inherited disorder that primarily affects boys, leading to progressive muscle degeneration and weakness. Currently, over 70,000 families in China are facing the challenges brought on by this condition, which has historically lacked effective treatment options. With the endorsement of AGAMREE, Santhera’s CEO Dario Eklund expressed optimism: 'This approval allows us to help those suffering from DMD, paving the way for better quality of life for many children in China.' Santhera is eager to collaborate with Sperogenix Therapeutics as they prepare to bring AGAMREE to market.

Collaborative Efforts to Push Forward

Mr. Yan Zhiyu, the Co-founder, Chairman, and CEO of Sperogenix Therapeutics, underscored the significance of this approval, acknowledging the government’s commitment to expedite the development of essential drugs for rare diseases. 'This milestone is a testament to our resolve to address the urgent needs of patient communities,' he emphasized, highlighting the urgency for accessible treatment.

AGAMREE's Unique Mechanism of Action

What sets AGAMREE apart from existing treatments is its unique mechanism of action. Unlike traditional corticosteroids, which often come with significant adverse effects, AGAMREE binds to the same receptor while modifying its downstream activity, thereby reducing safety concerns and potentially dissociating efficacy from steroid-associated side effects. This positions AGAMREE as a promising alternative for managing the condition effectively.

Promising Clinical Trial Results

The pivotal VISION-DMD study demonstrated that AGAMREE achieved key clinical endpoints, with a remarkable improvement in Time to Stand (TTSTAND) velocity compared to placebo. Alongside demonstrating efficacy, AGAMREE maintained a favorable safety profile, reinforcing its potential as a primary treatment option for DMD.

Future Steps for AGAMREE

Santhera operates under an exclusive collaboration with Sperogenix for AGAMREE’s commercialization in China. Following the approval, arrangements are in place for Sperogenix to pay royalties to Santhera, reflecting their shared commitment to advancing DMD treatment. The focus is now on accelerating access and distribution practices to ensure that patients can receive this vital therapy as quickly as possible.

About Santhera Pharmaceuticals

Santhera Pharmaceuticals (SIX: SANN) is dedicated to developing innovative therapies for rare neuromuscular conditions. AGAMREE stands at the forefront of their research initiatives, with the company committed to improving the lives of those affected by DMD. The approval of this therapy in China is a significant step forward in their mission to deliver cutting-edge treatment options.

Frequently Asked Questions

What is AGAMREE?

AGAMREE® (vamorolone) is a novel therapy developed by Santhera Pharmaceuticals for the treatment of Duchenne muscular dystrophy, designed to provide an effective treatment option with reduced safety concerns.

Who is affected by DMD?

Duchenne muscular dystrophy predominantly affects boys and manifests through progressive muscle degeneration, severely impacting mobility and overall health.

What does NMPA approval mean for AGAMREE?

The NMPA approval signifies that AGAMREE is authorized for treatment in patients aged four and older in China, making it the first approved therapy for DMD in the nation.

What are the expected benefits of AGAMREE?

AGAMREE aims to improve muscle function in DMD patients while minimizing side effects commonly associated with traditional steroid treatments, providing a new hope for better management of the disease.

How can patients access AGAMREE?

After its commercial launch, patients can access AGAMREE through medical channels established by Sperogenix Therapeutics in China, with further information to be announced as the product rolls out.

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