Qilu Pharmaceutical's Romiplostim N01, Rare Disease Drug Among Four Products Included in New National Reimbursement Drug List; Iruplinalk Tablet Expands Coverage

Nov 29, 2024 11:06 CST Updated 11:06

Qilu Pharmaceutical

Specialty Formulations and Active Pharmaceutical Ingredients (API) Developer

On November 28, the National Healthcare Security Administration announced the adjustment results of the 2024 National Basic Medical Insurance, Work-related Injury Insurance, and Maternity Insurance Drug Catalog. In this adjustment, a total of 91 drugs were newly added to the national medical insurance drug catalog, and 43 drugs were removed from the catalog.

Of the 117 off-list drugs participating in this year’s negotiations or bidding, 89 succeeded in the negotiation or bidding process, with a success rate of 76%. The price reduction was 63%, which is generally consistent with previous years.

Overall, among the 91 newly added drugs, the negotiation success rate for innovative drugs exceeded 90%, demonstrating unprecedented support from medical insurance for innovation. Notably, in the 2024 medical insurance negotiations, domestically produced innovative drugs performed exceptionally well, with multiple varieties successfully included in the national medical insurance catalog. The proportion of drugs from domestic companies exceeded 70%, reflecting that pharmaceutical innovation in China is in a phase of sustained rapid development.

In the seven years since the establishment of the National Healthcare Security Administration, the time it takes for new drugs to be included in health insurance has become increasingly shorter. In 2019, newly negotiated drugs took an average of 5.7 years from receiving approval to being included in the national health insurance. Since then, this process has continuously accelerated. By 2023, 57 drugs achieved "approval and inclusion in the same year" during the adjustment of the health insurance catalog. This trend has continued into 2024, with inclusion in health insurance in the same year as market launch becoming a major pathway in the commercialization of innovative drugs.

Whether it is the Class 1 innovative drug Iruak tablet developed by Qilu Pharmaceutical that entered the medical insurance in 2023, or the injectable Romiplostim N01 that was included in the medical insurance through negotiations in this year's directory update, both are representative of such new drugs.

The success of domestically produced innovative drugs in national healthcare negotiations not only reflects the country's support for pharmaceutical innovation but also provides strong assurance for the market promotion and patient usage of these drugs. As more innovative drugs are included in the national healthcare catalog, the financial burden on patients will be further reduced, while also helping to drive the rapid development of China’s pharmaceutical industry and enhance its innovation capabilities.

Included in the medical insurance in the year of listing, ushering in a new era of ITP patient treatment

The launch and smooth inclusion of Romiplostim N01 into the medical insurance system has brought more convenient, effective, and safer treatment options for patients with chronic primary immune thrombocytopenia (ITP), providing a more ideal solution for long-term management.

ITP is an acquired autoimmune bleeding disorder characterized by isolated peripheral thrombocytopenia without a clear cause. The number of patients in China is approximately 550,000, with the elderly population aged 60 and above being the high-risk group. Clinical manifestations of ITP vary significantly, ranging from asymptomatic thrombocytopenia, mucocutaneous bleeding, severe internal bleeding, to life-threatening intracranial hemorrhage. The risk of fatal bleeding in elderly patients is notably higher than in younger patients.

Statistics show that approximately 70%-80% of adult ITP patients have a chronic disease course. Currently, first-line treatments mainly include corticosteroids and intravenous immunoglobulin, but the long-term cure rate is only 20%-30%. Moreover, in severe cases, corticosteroids can temporarily increase platelet counts, but due to significant side effects, they cannot be used continuously, making it difficult to achieve long-term remission. Patients often experience recurrent episodes of the disease, indicating substantial unmet clinical needs.

On April 11 this year, Qilu Pharmaceutical's Class 3.4 biologic drug Romiplostim for Injection N01 (trade name: Ruili Sheng)^®) has been approved by the China National Medical Products Administration (NMPA) for adult patients (≥18 years old) with chronic immune thrombocytopenia (ITP) who have had an inadequate response to other treatments (e.g., corticosteroids, immunoglobulins). This is also the first domestically produced romiplostim injection approved for marketing in China.

As the second-generation long-acting thrombopoietin (TPO) receptor agonist, Romiplostim was awarded the "Best Biotechnology Product" by the Galien Award in 2009. Speaking of Qilu Pharmaceutical's layout in this area, it can be traced back to 2013. Eleven years ago, Qilu Pharmaceutical's QL0911 (Recombinant Human Thrombopoietin Peptidomimetic-Fc Fusion Protein for Injection) submitted a clinical trial application to the NMPA. After nearly ten years of clinical development, it was submitted for marketing approval in March 2022.

In December 2023, Qilu Pharmaceutical announced that the Phase III study results of Romiplostim Injection (QL0911) were published in the academic journal *Journal of Translational Internal Medicine* (English edition). This was a Phase III trial evaluating the efficacy and safety of QL0911 in adult patients with chronic primary immune thrombocytopenia, led by Professor Hou Ming from Qilu Hospital of Shandong University. The corresponding authors of the article are Professor Hou Ming and Professor Shi Yan from Qilu Hospital of Shandong University.

The study results showed that among the 216 enrolled patients (144 in the romiplostim group and 72 in the placebo group), during the 24-week double-blind period, 61.8% of the patients in the romiplostim group achieved a sustained platelet response (platelet count ≥50×10^9/L for 6 out of the last 8 weeks), while no patients in the placebo group achieved a sustained platelet response.

Moreover, the average number of weeks with platelet response (platelet count ≥50×10^9/L) during the 24-week double-blind period was 15.9 weeks for patients in the romiplostim group. In patients who had previously undergone splenectomy, the average number of weeks with platelet response was also 15.9 weeks, compared to only 1.9 weeks in the placebo group. The median time from the initial dose of 1 μg/kg romiplostim to the first platelet response (platelet count ≥50×10^9/L) was 3.29 weeks for patients in the romiplostim group.

Researchers believe that injectable Romiplostim shows significantly superior efficacy compared to the placebo group in clinical trials involving Chinese patients with chronic adult primary immune thrombocytopenia. It rapidly increases and maintains platelet counts, demonstrates good tolerability, and provides an effective and accessible treatment option for Chinese patients with chronic primary immune thrombocytopenia. The once-a-week dosing frequency also greatly improves patient compliance.

It is reported that currently, clinical experts in China are actively exploring the clinical efficacy of Romiplostim in multiple disease areas, such as severe ITP, pediatric ITP, first-line ITP treatment combined with glucocorticoids, chemotherapy-induced thrombocytopenia (CTIT), aplastic anemia (AA), and thrombocytopenia after transplantation (HSCT). Meanwhile, a randomized controlled trial of Romiplostim for CTIT treatment is also underway, which is expected to provide a new treatment option for CTIT.

Represented by Iluak, innovative drugs continue to "advance and capture territories"

In June 2023, Qilu Pharmaceutical's innovative drug Irulak was approved for marketing by the National Medical Products Administration. It is Qilu’s first self-owned intellectual property Class 1 innovative drug. In January this year,Approved again for monotherapy in patients with anaplastic lymphoma kinase (ALK)-positive locally advanced or metastatic non-small cell lung cancer (NSCLC), adding a first-line indication, fully covering ALK-positive locally advanced or metastatic NSCLC patients.

For Irulak, which entered the medical insurance catalog in 2023, the "main task" of this year's medical insurance negotiation is to expand the indications covered by medical insurance payments.

From the list of the new edition of the medical insurance directory, the medical insurance payment scope for Irulak has been changed from "limited to patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) who are positive for anaplastic lymphoma kinase (ALK), have progressed after previous treatment with Crizotinib, or are intolerant to Crizotinib" to "limited to patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) who are positive for anaplastic lymphoma kinase (ALK)."

From the R&D project initiation, determination of candidate compounds, pharmaceutical research, preclinical studies to Phase I, II, and III clinical trials, and finally to its approval for marketing, it took Iruplak 10 years, making its success a "decade-long sharpening of a sword." This also marks a significant step forward for Qilu Pharmaceutical in the field of innovative drugs. To date, Iruplak has been selected as a major achievement of the national "Major New Drug Creation" science and technology project, with its clinical data presented at top academic conferences both in China and internationally, including WCLC, ASCO, ESMO, and CSCO.

Iluak is not only the beginning of Qilu Pharmaceutical's innovative R&D entering the harvest period, but also a microcosm of Qilu's efforts in overcoming difficulties in innovative drug development.

Over the past decade, Qilu Pharmaceutical's R&D talent team has grown from over 1,000 to more than 5,000 people. At the same time, the company has consistently maintained an R&D investment intensity of 10% of sales revenue, with a total R&D investment of 13.68 billion yuan in the past five years and an annual growth rate exceeding 20%. Since 2014, Qilu Pharmaceutical has successively established six major R&D centers in Seattle, San Francisco, and Boston in the United States, as well as in Shanghai, its headquarters in Jinan, and Hainan. These centers widely attract scientific research talents from home and abroad, aggregate high-quality intellectual resources globally, seize the commanding heights of innovative drug R&D, and form a complete innovation development system covering the early discovery, development, and industrialization of chemical drugs and biotech drugs. In significant unmet disease areas such as cancer, infectious diseases, autoimmune diseases, and metabolic disorders, the company continues to develop innovative drugs.

Among them, Mabpair, globally and independently innovated by Qilu Pharmaceutical^®Technology Platform: The same cell line can express two different antibodies, thereby covering two targets and achieving a叠加 of therapeutic effects. The platform's first next-generation combination antibody for cancer immunotherapy is the injection of Aipaloli Tovorali (trade name: Qibean).^®) was approved for marketing in September this year, and the innovative achievements of Qilu Pharmaceutical continue to materialize.Currently, Qilu Pharmaceutical has more than 80 innovative drugs in the research and development stage.It is foreseeable that in the next few years, Qilu Pharmaceutical will launch more innovative drugs to address unmet clinical needs.

Great Medical Dedication: Enhancing the Accessibility and Affordability of Medications for Patients

Not only Romiplostim Injection N01 and Iruka Tablets, but also Qilu Pharmaceutical's Tafamidis Meglumine Soft Capsules and Metformin Empagliflozin Tablets (Ⅵ) were dynamically adjusted into the medical insurance catalog through competitive bidding during this year's adjustment.

These two products were both recently approved for marketing in June this year and are both the first domestic generics in China (including those approved on the same day).Tafamidis Meglumine Soft Capsules for the treatment of adultsPatients with Stage I symptoms of Transthyretin Amyloidosis Polyneuropathy (ATTR-PN),Can effectively delay peripheral nerve function damage. ATTR-PN is a rare, hereditary, progressively worsening, and fatal disease., a rare hereditary, progressively worsening, and fatal disease that predominantly occurs in people aged 30-40, is irreversible and poses a serious threat to patients' lives.The original research product of Tafamidis Meglumine Soft Capsules was launched in China in 2020, with high treatment costs.To meet the needs of clinical patients, Qilu Pharmaceutical Co., Ltd. has actively explored solutions to overcome the "last mile" barrier in providing medications for rare disease patients. The company initiated the development of Tafamidis Meglumine Soft Capsules, receiving marketing approval in June 2024.With the successful inclusion of Vyndaqel (Tafamidis Meglumine Soft Capsules) in the medical insurance, the financial burden on ATTR-PN patients will be greatly reduced, allowing more patients with rare diseases to receive more standardized treatment.At the same time, the support from China's medical insurance will effectively promote the medical industry's clinical understanding and diagnostic treatment level for rare diseases, especially ATTR-PN.With the dual support of being the first generic drug and entering the medical insurance, both Tafamidis Methylglucamine Soft Capsules and Metformin Empagliflozin Tablets (VI) are expected to accelerate patient access.

In recent years, China's chemical generic drug market has undergone a series of policy adjustments, from the full implementation of consistency evaluations to the manifestation of the original research substitution effect under bulk procurement, leading the market into a continuous adjustment cycle. Data from "China Generic Drug Development Report (2023 Edition)" shows that in 2023, the scale of China’s chemical generic drug market reached 892.3 billion yuan, a year-on-year increase of 2%.

On multiple public occasions, the relevant person in charge of Qilu Pharmaceutical has emphasized that the company's original intention is to "enable ordinary people to access and afford high-quality domestically produced medicines." Qilu has adhered to the values of "Excellence in medicine, serving the nation and society, with a prosperous country and strong factories ensuring our happy homes" for over forty years. In response to the still unmet clinical medication needs in China, on one hand, the company develops innovative drugs targeting serious diseases to enhance the competitiveness of domestic pharmaceuticals; on the other hand, it spares no effort in continuously researching and developing high-quality generic drugs to address issues of drug accessibility and affordability.

According to Qilu Pharmaceutical, the company's various products have benefited more than 100 countries and regions worldwide. For many years, it has remained the only pharmaceutical enterprise in China that simultaneously exports formulations to the regulated markets of Europe, the United States, the United Kingdom, Japan, Australia, and Canada, with 27 products ranking first in local market share. Cisplatin Injection, using domestically marketed packaging, supplies the U.S. as a shortage drug; Ranibizumab received approval for marketing in the EU and UK, becoming the first ophthalmic biologic drug from China to enter overseas markets. With extraordinary innovation capabilities, Qilu Pharmaceutical has set its sights on cutting-edge technology, carving out new paths in the pharmaceutical field with remarkable foresight, and repeatedly seizing opportunities.

References:

1. https://mp.weixin.qq.com/s/Ost266Kkjnap0hHnSTKf9A

2. https://mp.weixin.qq.com/s/_hFCt6sMDNsUJbTx7-DmXQ

First Review | Huang Jia

Second Review | Li Fangchen

Third Review | Li Jingzhi

Featured Recommendations