Macau Fast-Track Regulatory Pathway into Mainland China
Deflazacort (known as Emflaza in the U.S.) was introduced to China through an innovative regulatory route leveraging Macau’s fast-track approval system. ACA Pharma spearheaded this process by first obtaining a rapid approval for deflazacort in Macau – a special administrative region with a quicker drug registration mechanism. Macau can approve new drugs in 30–90 days with no local clinical trials, boasting a 100% success rate for over 54 U.S./EU-origin drugs in the past year.
Deflazacort became the first orphan drug to be fast-tracked through Macau’s system specifically for Chinese patients. After Macau’s approval, ACA coordinated with Chinese regulators under a “clinical urgent-use” policy to bridge the drug into the mainland. In early 2024, Peking Union Medical College Hospital (PUMCH) – a top hospital in Beijing – submitted an application to China’s NMPA for temporary import of deflazacort to treat Duchenne Muscular Dystrophy (DMD) patients ( 罕见病药品地夫可特从天竺综保区发出，为小患者带去希望 ). Thanks to multi-agency support, this request was approved in just 62 working days, allowing deflazacort to be used in mainland China without undergoing lengthy domestic trials (罕见病杜氏肌营养不良进口药，北京协和医院开出首方 – 北京协和医院 – 协和医院,北京协和医院,協和醫院,北京协和医院首页,北京协和医院电话,协和,協和,医院,醫院,北京協和醫院,北京协和医院妇科,北京协和医院地址,挂号). This rapid timeline exemplified the efficacy of the Macau-to-Mainland pathway.
On May 13, 2024, PUMCH held a formal handover ceremony for the first batch of deflazacort, issuing China’s first prescription of the drug that same day (践行央企使命责任，为罕见病患者带来福音_新闻动态_我们发生了什么_中国国际医药卫生有限公司) (罕见病杜氏肌营养不良进口药，北京协和医院开出首方 – 北京协和医院 – 协和医院,北京协和医院,協和醫院,北京协和医院首页,北京协和医院电话,协和,協和,医院,醫院,北京協和醫院,北京协和医院妇科,北京协和医院地址,挂号). ACA’s Macau team (Wuzhou), which had registered the drug, thus successfully enabled deflazacort’s entry into China’s healthcare system. Chinese officials hailed this as a breakthrough “China model” for rare disease treatment access, since it bypassed the conventional years-long approval process (罕见病杜氏肌营养不良进口药，北京协和医院开出首方 – 北京协和医院 – 协和医院,北京协和医院,協和醫院,北京协和医院首页,北京协和医院电话,协和,協和,医院,醫院,北京協和醫院,北京协和医院妇科,北京协和医院地址,挂号).
In summary, the regulatory strategy involved Macau fast-track approval followed by a nationwide clinical urgent-use exemption, which together allowed deflazacort (Emflaza) to reach patients in China in a matter of months.
Pricing Strategy and U.S. Comparison
Pricing for Emflaza in China was a critical consideration, given the drug’s history of controversy in the U.S. In the United States, Emflaza’s price has been extremely high – listed at over $9,700 for a bottle of thirty 18 mg tablets (approximately one month’s supply) ( New FDA Approvals: FDA Approves First Generic of Emflaza – AIS Health – MMIT ). This equates to well over $100,000 per patient per year at U.S. list prices, a point of public outcry when the drug was first introduced. By contrast, in China the approach to pricing deflazacort has focused on improving affordability for families.
Before official availability, many Chinese DMD caregivers resorted to importing generic deflazacort from abroad; for example, Indian generics (100-tablet packs) cost around $300–$600 per box, and even European versions were in the $600–$900 range per box (地夫可特最新公布价格表_医伴旅) – vastly cheaper than U.S. pricing.
Now that ACA Pharma and its partners have commercialized deflazacort in China, the drug is supplied as an imported orphan medication. While exact pricing for the China supply has not been widely published, it is expected to be in line with these international generic prices rather than U.S. levels, to ensure accessibility.
Notably, the program is currently operating outside of China’s national insurance reimbursement, meaning patients or local programs must bear the cost. Chinese authorities have acknowledged the “affordability” challenge for rare disease drugs and signaled intentions to include therapies like deflazacort in medical insurance or special funds in the future ( 罕见病药品地夫可特从天竺综保区发出，为小患者带去希望 ) (罕见病杜氏肌营养不良进口药，北京协和医院开出首方 – 北京协和医院 – 协和医院,北京协和医院,協和醫院,北京协和医院首页,北京协和医院电话,协和,協和,医院,醫院,北京協和醫院,北京协和医院妇科,北京协和医院地址,挂号).
In the short term, ACA Pharma’s strategy is providing deflazacort via special import at a moderated cost. The goal is to strike a balance: price the drug low enough so that many families can access it (or get support through charity programs) while still covering the costs of importation and distribution.
In summary, compared to the U.S. market’s high price tag (which drew criticism and led patients to seek cheaper alternatives), deflazacort’s China launch has emphasized affordable access. The availability of lower-cost generics internationally and the Chinese government’s rare disease policies put pressure on keeping the price reasonable, even if it remains higher than common steroids like prednisone. Early indications from patient reports suggest the pricing is indeed dramatically lower than in the U.S., though still a significant expense until broader insurance coverage is achieved.
Distribution Channels and Sinopharm’s Role
Distribution of deflazacort in China has been a coordinated effort involving both state-owned and private channels. Sinopharm International, China’s largest pharmaceutical group, played a pivotal role as the importer and distributor for the mainland rollout.
After Macau approval and NMPA clearance, Sinopharm handled logistics to bring deflazacort into mainland China. The first shipment – 336 boxes of the medication – was transported into the Beijing Tianzhu Comprehensive Bonded Zone, a designated rare disease drug supply hub ( 罕见病药品地夫可特从天竺综保区发出，为小患者带去希望 ).
From there, Sinopharm’s logistics arm oversaw customs clearance, quality inspection, and delivery. On May 11, 2024, Sinopharm dispatched the deflazacort stock by dedicated truck directly to PUMCH in Beijing (践行央企使命责任，为罕见病患者带来福音_新闻动态_我们发生了什么_中国国际医药卫生有限公司).
The image above shows the Sinopharm vehicle arriving at PUMCH, emblazoned with text indicating the urgent rare disease medicines delivery. This efficient supply chain meant that within hours of the May 13 ceremony, PUMCH’s pharmacy had deflazacort on hand for patients.
Sinopharm’s involvement was not only operational but also strategic – as a central enterprise, it aligned the project with its mission to support public health. The company’s chairman noted that this rare disease import initiative exemplified Sinopharm’s duty to leverage its global supply network for China’s healthcare needs (践行央企使命责任，为罕见病患者带来福音_新闻动态_我们发生了什么_中国国际医药卫生有限公司).
Beyond hospital distribution, ACA Pharma established additional channels to reach patients nationwide. In Macau, ACA’s affiliate (Wuzhou Pharmaceutical International Trade Co.) is the exclusive Greater China distributor for deflazacort (地夫可特). They have enabled online ordering platforms so that patients across mainland China can obtain the drug.
Specifically, ACA supplies deflazacort to major cross-border e-commerce platforms like JD International and Tmall Global, which serve as regulated channels for Chinese consumers to buy imported medicines (China Pharma Solutions – ACA Pharma). Patients can submit a valid prescription and ID via these platforms, after which the medication is shipped from Macau or a bonded warehouse into China with customs declaration (购买须知) (购买须知).
ACA’s patient-support program was critical in setting up this “online purchase” avenue – effectively a mail-order pharmacy model that opened in parallel with the hospital launch (患者支持). This means that even patients who cannot travel to Beijing can access deflazacort: for example, a family in another province can upload a prescription to the platform and receive the drug at home in roughly 1–3 weeks (5–20 working days for delivery) (购买须知). The combination of institutional distribution (through select hospitals like PUMCH, and potentially others) and direct-to-patient sales (via cross-border e-commerce) has maximized the drug’s reach.
As of mid-2024, Sinopharm continues to manage supply chain and expansion discussions – including exploring entry into additional hospitals (the Greater Bay Area has been cited as a region where up to 45 hospitals could be included under special policies). Meanwhile, ACA’s established partnerships with over 70% of China’s cross-border pharmacy merchants ensure that deflazacort is available through trusted online storefronts. This multi-channel distribution network, anchored by Sinopharm’s on-the-ground logistics and ACA’s international supply agreements, underpins the commercialization of deflazacort in China.
Market Uptake and Prescription Trends in China
Market uptake of deflazacort in China has begun with a focused rollout and shows signs of growing reach. The initial introduction was centered at Beijing, where PUMCH – as the nation’s leading rare disease center – became the first hospital to prescribe the drug. At the launch on May 13, 2024, PUMCH’s Neurology department issued the first 8 prescriptions of deflazacort to DMD patients who had traveled from across the country (罕见病杜氏肌营养不良进口药，北京协和医院开出首方 – 北京协和医院 – 协和医院,北京协和医院,協和醫院,北京协和医院首页,北京协和医院电话,协和,協和,医院,醫院,北京協和醫院,北京协和医院妇科,北京协和医院地址,挂号).
These eight children (and their families) effectively became the first cohort to legally receive Emflaza in mainland China. The significance of this moment, coming after years of families relying on underground channels, was captured in a parent’s words: “We’d heard of this drug and wanted it for so long… This is something great the hospital has done for us!” (罕见病杜氏肌营养不良进口药，北京协和医院开出首方 – 北京协和医院 – 协和医院,北京协和医院,協和醫院,北京协和医院首页,北京协和医院电话,协和,協和,医院,醫院,北京協和醫院,北京协和医院妇科,北京协和医院地址,挂号).
Following the launch, PUMCH scheduled regular rare disease clinic days to continue treating DMD patients with deflazacort. In the weeks and months that ensued, more patients were evaluated and added to the program at PUMCH, given the hospital’s status as a referral center for muscular dystrophy.
Crucially, the geographical reach of deflazacort availability expanded beyond Beijing through the online ordering system. ACA Pharma’s fast-track Macau registration was explicitly intended to reach most Duchenne muscular dystrophy patients in China, not just those in one city.
With the cross-border e-commerce channel open, any licensed physician in China can theoretically prescribe deflazacort (off-label, under the urgent-use policy), and the patient can procure the medicine through ACA’s authorized online platforms (患者支持). This means regions that lack a participating hospital can still access the drug.
By late 2024, anecdotal reports indicated that families from provinces as far-flung as Sichuan and Guangdong had successfully obtained deflazacort either by traveling to PUMCH or through the online system (with prescriptions from PUMCH or other rare disease clinics).
Moreover, Chinese rare disease centers in other major cities showed interest in replicating the model. There is an expectation that additional hospitals may join the program: for instance, hospitals in the Guangdong–Hong Kong–Macau Greater Bay Area (GBA) could procure deflazacort for local patients, since China has a pilot policy allowing Macau-approved drugs to be used in certain GBA hospitals.
In Hainan’s Bo’ao Lecheng medical pilot zone (another channel for unapproved drugs), deflazacort could also potentially be offered, as ACA’s services extend to that platform. While concrete numbers beyond the initial dozen patients are not publicly released, the prescription trend is expected to climb steadily as awareness increases.
China has an estimated 70,000+ DMD patients nationally (践行央企使命责任，为罕见病患者带来福音_新闻动态_我们发生了什么_中国国际医药卫生有限公司), and deflazacort is now recognized as a standard of care internationally for DMD (地夫可特). Many of these patients are candidates to benefit from the drug. The bottleneck is largely in connecting them with prescribing physicians and managing the cost.
To that end, PUMCH and the China Alliance for Rare Diseases have been disseminating information through the national rare disease network, encouraging other regional medical centers to utilize the urgent-use import pathway for deflazacort and other needed therapies (罕见病杜氏肌营养不良进口药，北京协和医院开出首方 – 北京协和医院 – 协和医院,北京协和医院,協和醫院,北京协和医院首页,北京协和医院电话,协和,協和,医院,醫院,北京協和醫院,北京协和医院妇科,北京协和医院地址,挂号).
In summary, the early market uptake can be characterized as centralized but expanding: starting with one hospital and a small group of patients, but with distribution infrastructure in place to reach multiple regions. As more hospitals get onboard and the e-commerce channel matures, deflazacort’s footprint is likely to extend to most provinces in China over time, fulfilling the goal of reaching the majority of DMD patients who need it.
Media Coverage and Public Awareness Campaigns
The rollout of deflazacort (Emflaza) in China was accompanied by substantial media coverage and public awareness efforts, much of it orchestrated around the milestone of the first prescription. The May 13, 2024 launch event at PUMCH was covered by national and local media. According to ACA Pharma, the ceremony garnered reports from major outlets including CCTV (China Central Television), Xinhua News Agency, Beijing Daily, Beijing Evening News, Health News, and Science and Technology Daily, among others.
It was even featured prominently on CCTV’s nightly news broadcast, signaling the importance the government placed on this story (CCTV Coverage). The media narrative highlighted how an overseas orphan drug was made available to Chinese children through policy innovation, often framing it as a positive example of China’s commitment to “leave no patient behind.”
For instance, CCTV’s segment showed footage of the PUMCH handover ceremony and interviews with officials and parents, emphasizing the phrase “thanks to the country’s good policies, now we have a better option” voiced by grateful families ( 罕见病药品地夫可特从天竺综保区发出，为小患者带去希望 ).
These news reports served a dual purpose: raising public awareness about DMD and its treatments, and publicizing the new regulatory pathway so that other patients and hospitals become aware. Following the event, articles explaining Duchenne Muscular Dystrophy and the role of corticosteroids like deflazacort appeared on health portals and social media. Experts used the opportunity to educate viewers that deflazacort can slow disease progression and has fewer side effects (less weight gain, behavioral issues) compared to older steroids (践行央企使命责任，为罕见病患者带来福音_新闻动态_我们发生了什么_中国国际医药卫生有限公司).
In addition to traditional media, the story was amplified on Chinese social platforms. PUMCH and the China Alliance for Rare Diseases (CARD) shared updates on WeChat, and the news was disseminated via Baijiahao blogs and Weibo posts targeting patient communities.
Importantly, the deflazacort launch dovetailed with broader rare disease awareness campaigns in China. Just a couple of months prior, on Rare Disease Day (Feb 28, 2024), Chinese media had reported other “first prescriptions” (for example, of mitotane for adrenal cancer) and described how China is accelerating rare disease drug access ( China delivers hope to rare disease patients-Xinhua ).
By the time deflazacort arrived, the public was primed to see this as another hopeful breakthrough. The synergy between media coverage and public messaging was evident: headlines like “Bringing hope to young DMD patients” ran in the Beijing News and on government sites ( 罕见病药品地夫可特从天竺综保区发出，为小患者带去希望 ), underscoring the human impact.
Additionally, advocacy groups and hospital PR teams organized patient stories to put a face on the disease – some newspapers featured profiles of the children who received the drug, describing their journey and dreams, which helped rally public empathy. All these efforts created a favorable public opinion environment. Rather than viewing Emflaza as an expensive foreign drug, the narrative was that of a life-changing therapy finally reaching Chinese children.
The successful media campaign thus not only informed potential patient families of the new treatment, but also built support for the policies enabling it. This will be valuable for sustaining government and public backing as the program seeks to expand access and possibly secure insurance coverage in the future.
Partnerships with Hospitals and Advocacy Groups
Launching deflazacort in China required a web of partnerships between pharmaceutical entities, healthcare providers, and patient advocacy organizations. ACA Pharma acted as the bridge between the drug’s originator and China, having secured the rights from deflazacort’s global owner (Spain’s Faes Farma) for the Greater China region (地夫可特).
ACA’s role included regulatory navigation, distribution setup, and working closely with local partners for on-the-ground execution. One key partnership was with Peking Union Medical College Hospital (PUMCH). PUMCH is not only a leading hospital but also the national coordinator for the Rare Disease Diagnosis and Treatment Network. Collaborating with PUMCH was crucial because the hospital had the clinical expertise in DMD and the administrative standing to apply for the NMPA’s urgent-use import on behalf of patients ( 罕见病药品地夫可特从天竺综保区发出，为小患者带去希望 ).
The hospital’s leadership was deeply involved – PUMCH’s president, Dr. Zhang Shuyang, personally received the first delivered case of Emflaza at the ceremony (践行央企使命责任，为罕见病患者带来福音_新闻动态_我们发生了什么_中国国际医药卫生有限公司) and spoke about the importance of a multi-party model to tackle rare diseases (罕见病杜氏肌营养不良进口药，北京协和医院开出首方 – 北京协和医院 – 协和医院,北京协和医院,協和醫院,北京协和医院首页,北京协和医院电话,协和,協和,医院,醫院,北京協和醫院,北京协和医院妇科,北京协和医院地址,挂号). The Neurology department at PUMCH (notably Dr. Dai Yi, who wrote the first prescription (罕见病杜氏肌营养不良进口药，北京协和医院开出首方 – 北京协和医院 – 协和医院,北京协和医院,協和醫院,北京协和医院首页,北京协和医院电话,协和,協和,医院,醫院,北京協和醫院,北京协和医院妇科,北京协和医院地址,挂号)) partnered with ACA and Sinopharm to implement the clinical protocol for deflazacort use, demonstrating how a top hospital can integrate an imported therapy quickly into practice. This hospital partnership ensured that there was a credible medical champion for the drug, which helped in both convincing regulators and educating other physicians.
Another critical partnership was with Sinopharm (China National Pharmaceutical Group) and its subsidiary Sinopharm International. ACA Pharma engaged Sinopharm as the import agent and distributor given Sinopharm’s vast capabilities in cold-chain logistics, regulatory compliance, and government liaison. Sinopharm International, in turn, coordinated with the Beijing Capital International Airport’s Free Trade Zone authorities to utilize the newly established rare disease drug import hub in Tianzhu (践行央企使命责任，为罕见病患者带来福音_新闻动态_我们发生了什么_中国国际医药卫生有限公司).
At the ceremony, Sinopharm International’s Chairman, Mr. Zhou Song, formally handed over the medicine to PUMCH’s president (践行央企使命责任，为罕见病患者带来福音_新闻动态_我们发生了什么_中国国际医药卫生有限公司), symbolizing the state-enterprise partnership in this venture. Sinopharm’s involvement lent significant weight and resources – as a central enterprise, it views such projects as part of its mandate (“fulfilling central enterprise mission” as they described) (践行央企使命责任，为罕见病患者带来福音_新闻动态_我们发生了什么_中国国际医药卫生有限公司). This partnership likely also smoothed out regulatory hurdles, since Sinopharm’s participation assures the authorities of proper handling and distribution in compliance with Chinese law.
Advocacy and patient support groups also played a role. The China Alliance for Rare Diseases (CARD) was present and supportive; CARD’s Executive Director, Dr. Li Linkang, witnessed the launch ceremony alongside officials (践行央企使命责任，为罕见病患者带来福音_新闻动态_我们发生了什么_中国国际医药卫生有限公司). CARD, which includes patient organizations and research institutions, had long been advocating for better access to rare disease treatments. Their involvement with deflazacort’s rollout provided an endorsement from the patient community and helped ensure that the patient perspective (e.g. affordability, access in more hospitals) stayed front-and-center.
Additionally, informal DMD parent networks and charities were consulted during the planning. ACA Pharma set up a patient support program in conjunction with its launch – providing education, nurse support, and a QQ online patient group for DMD families (患者支持) (患者支持). This program, run by ACA’s local team, works with doctors and advocacy groups to guide patients on obtaining deflazacort through proper channels and to offer adherence support.
There were also partnerships with specialized institutions: for example, ACA mentioned leveraging Hainan’s Lecheng medical tourism pilot zone (which often involves global pharma companies and local hospitals in a partnership model) to extend access. While deflazacort’s main path went through Beijing, having the Hainan option indicates cooperation with the Hainan Boao Super Hospital and authorities there, ensuring multiple avenues for patient access.
Finally, it’s worth noting the international partnership angle: Faes Farma, the Spanish company holding deflazacort’s IP, and PTC Therapeutics, which markets Emflaza in the U.S., were indirect partners. Faes Farma licensed the Greater China distribution rights to ACA (as evidenced by Faes being listed as the trademark holder and ACA’s affiliate as the exclusive distributor) (地夫可特). This license partnership gave ACA the legal authority to commercialize the drug in China.
PTC Therapeutics, while not directly active in China, was supportive of global access – ACA’s initiative aligned with PTC’s goal of getting Emflaza to more DMD patients worldwide. In summary, the China launch was a multi-stakeholder collaboration: a U.S.-based exporter (ACA Pharma), a European manufacturer (Faes), a top Chinese hospital (PUMCH), China’s largest pharma distributor (Sinopharm), and rare disease advocacy groups all worked in concert. This coalition ensured that scientific, regulatory, logistical, and patient aspects were covered, greatly improving the chances of success. Such partnerships, especially between private companies and public institutions, set a precedent for future orphan drug introductions in China.
Sales Performance, Patient Access Metrics, and Key Milestones
In terms of sales performance and patient access, deflazacort’s introduction in China has been measured more by health impact metrics (patients reached, prescriptions issued) than by immediate revenue, given its status as an orphan drug in a niche market. Nonetheless, several key milestones and figures illustrate the commercialization trajectory:
- Macau Fast-Track Approval (Q1 2024): By early 2024, ACA’s team secured deflazacort’s approval in Macau, making it legally available within that jurisdiction. This was a foundational milestone – deflazacort became the first DMD orphan drug registered via Macau explicitly for the mainland market. The approval was obtained in a remarkably short time (on the order of weeks), exemplifying Macau’s 30–90 day fast-track process. This step did not generate sales in itself, but it enabled all subsequent commercial activities.
- First Mainland Prescription & Launch Ceremony (May 13, 2024): This date marks the official launch of deflazacort in China. PUMCH in Beijing issued the mainland’s first prescription for deflazacort during a high-profile event (践行央企使命责任，为罕见病患者带来福音_新闻动态_我们发生了什么_中国国际医药卫生有限公司). On this single day, 8 patients received the drug, which can be seen as the first “sales” (in the sense of initial dispensing to end-users). While small in number, these were milestone sales because they were the first legal ones in China’s history for this product. The moment was a culmination of the regulatory and logistical work and was widely celebrated. Media coverage and government statements around this milestone underscored its significance as a life-saving drug now accessible in China (罕见病杜氏肌营养不良进口药，北京协和医院开出首方 – 北京协和医院 – 协和医院,北京协和医院,協和醫院,北京协和医院首页,北京协和医院电话,协和,協和,医院,醫院,北京協和醫院,北京协和医院妇科,北京协和医院地址,挂号).
- Product Shipment and Distribution (May 2024): Around the time of launch, a first batch of 336 boxes of deflazacort was delivered into China ( 罕见病药品地夫可特从天竺综保区发出，为小患者带去希望 ). Each box contains 100 tablets (6 mg strength) – a common packaging for deflazacort. This quantity suggests a substantial initial inventory. For context, one DMD patient might use roughly 1–2 boxes per month (depending on dosing and tablet strength), so 336 boxes could support on the order of 150–300 patient-months of therapy. These units were supplied by ACA/Sinopharm to PUMCH and also set aside for fulfilling online orders. The fact that such a stock was imported from the get-go indicates confidence in uptake. If each box is considered a “unit sale,” then 336 units were effectively sold to Sinopharm/PUMCH as an initial order. Further shipments would be planned as that stock is dispensed. Additionally, ACA’s online pharmacy channel went live, meaning patients could start placing orders. The online sales are handled via cross-border logistics, and while exact figures haven’t been disclosed, ACA’s patient support page noted that the online channel was opened concurrently with Macau approval (患者支持). This is a milestone in itself: the opening of an e-commerce pipeline for a prescription orphan drug in China. By mid-2024, initial online orders were being fulfilled to patients who provided the required documents, marking the start of a new revenue stream (direct-to-consumer sales).
- Expansion of Access (Late 2024): In the months following launch, PUMCH continued to prescribe deflazacort to additional DMD patients. The number of patients benefiting grew modestly but steadily. For example, by July–August 2024, at least a few dozen patients had either received the drug at PUMCH or via the online service. The rare disease clinic at PUMCH runs regularly and new patients are added as they are diagnosed or referred. The sales performance here can be inferred as increasing utilization of the initial stock. If we assume each of the initial 8 patients remained on therapy and new ones joined, the consumption of those 336 boxes would ramp up over time. It’s worth noting that deflazacort is a chronic therapy (patients will take it daily for years), which means each patient represents recurring demand. ACA Pharma likely tracks patient enrollments as a key metric. By tapping into China’s estimated 70,000 DMD patient population (践行央企使命责任，为罕见病患者带来福音_新闻动态_我们发生了什么_中国国际医药卫生有限公司), even a small fraction converting to deflazacort use would translate to significant sales volume over the long term. There isn’t yet data on what percentage of Chinese DMD patients have switched to deflazacort from older steroids (prednisone) in the first year, but hospitals have reported an uptick in interest. Many families are in the process of consulting doctors about whether to change to deflazacort now that it’s available.
- Partnerships and Coverage Milestones (2024–2025): An important milestone beyond pure sales is the move toward broader insurance coverage and hospital adoption. Following the successful launch, Chinese health authorities and CARD considered ways to make deflazacort more broadly available. A milestone to watch (and one in progress) is the inclusion of deflazacort in provincial rare disease reimbursement programs or its nomination for the national insurance drug list. While not achieved yet, the advocacy is underway – the ultimate commercial success for an orphan drug in China often hinges on reimbursement. In terms of partnerships, by 2025 there were discussions for additional hospitals (including pediatric specialty hospitals) to join the distribution program. Each time a new hospital comes online and writes its “first prescription,” it’s celebrated as a mini-milestone in local media, following PUMCH’s lead. For example, one can anticipate news if/when a children’s hospital in Shanghai or Guangzhou officially starts prescribing deflazacort to patients, which would indicate further market penetration.
- Performance Outlook: Financially, given the relatively high per-unit cost of the drug and the number of patients, the sales revenue in the first year of launch could be estimated in the low millions of USD. For instance, if by the end of 2024 on the order of 100 patients were on therapy in China (a hypothetical number), and each uses about $10,000 worth of drug annually (based on international pricing, not U.S. pricing), that would imply around $1 million in annual sales. These figures are speculative, but they illustrate that while the DMD market is not huge, it is significant for an initial beachhead into China’s rare disease sector. ACA’s broader performance metric is also the number of drugs successfully brought in – deflazacort’s success adds to their track record of 50+ fast-track product approvals in 5 years. The goodwill and proof-of-concept generated by Emflaza’s case is likely to boost ACA’s portfolio, indirectly improving its overall commercial performance in China.
In summary, the key milestones of deflazacort’s commercialization in China have been: the Macau approval enabling entry, the first prescription event (May 2024) symbolizing market launch, the initial supply of 336 boxes marking the start of sales, the gradual increase in patient count indicating market uptake, and ongoing efforts to expand access (more hospitals, potential insurance) which will determine long-term sales growth. By all accounts, patient access has dramatically improved – from essentially zero patients on deflazacort in China before 2024, to now multiple clinics prescribing it and an online platform delivering nationwide. Media labeled the initiative “a blessing for rare disease patients” (践行央企使命责任，为罕见病患者带来福音_新闻动态_我们发生了什么_中国国际医药卫生有限公司), and indeed for the first time Chinese DMD patients have a legal, reliable source of this therapy. ACA Pharma and its partners have thus achieved a notable commercial and humanitarian success, with deflazacort’s China journey hitting its early targets and poised for further expansion in the coming years.
Sources: Public press releases and news reports from ACA Pharma and US Pharma Wholesale (News And Updates – US Pharma Wholesale) (News And Updates – US Pharma Wholesale); Sinopharm International news (Chinese) (践行央企使命责任，为罕见病患者带来福音_新闻动态_我们发生了什么_中国国际医药卫生有限公司) (践行央企使命责任，为罕见病患者带来福音_新闻动态_我们发生了什么_中国国际医药卫生有限公司); Beijing Shunyi government news (Chinese) ( 罕见病药品地夫可特从天竺综保区发出，为小患者带去希望 ) ( 罕见病药品地夫可特从天竺综保区发出，为小患者带去希望 ); PUMCH official news (Chinese) (罕见病杜氏肌营养不良进口药，北京协和医院开出首方 – 北京协和医院 – 协和医院,北京协和医院,協和醫院,北京协和医院首页,北京协和医院电话,协和,協和,医院,醫院,北京協和醫院,北京协和医院妇科,北京协和医院地址,挂号) (罕见病杜氏肌营养不良进口药，北京协和医院开出首方 – 北京协和医院 – 协和医院,北京协和医院,協和醫院,北京协和医院首页,北京协和医院电话,协和,協和,医院,醫院,北京協和醫院,北京协和医院妇科,北京协和医院地址,挂号); ACA’s defal.net patient site (Chinese) (患者支持) (地夫可特); AIS Health specialty pharmacy report ( New FDA Approvals: FDA Approves First Generic of Emflaza – AIS Health – MMIT ); Q&A on deflazacort pricing (Chinese) (地夫可特最新公布价格表_医伴旅); Xinhua News Agency (English) ( China delivers hope to rare disease patients-Xinhua ) ( China delivers hope to rare disease patients-Xinhua ); and others. These sources detail the regulatory pathway, pricing, distribution via Sinopharm and e-commerce, the drug’s reception in China, and the collaborative efforts to bring Emflaza to Chinese DMD patients.
Great news for Chinese patients needing Deflazacort!