Global Drug Approvals/R&D UpdatesGlobal New Drug ApprovalsAccording to the data analysis by PharmaDJ, during this statistical period (September 21-27, 2024), a total of 13 new drugs were approved for marketing globally (excluding China). Among them, there were 5 NDA approvals, 3 BLA approvals, 1 new compound approval, and 4 new indication approvals. Compared with the previous statistical period, there are 2 more drug approvals in this cycle.
On September 23, Ipsen's Kayfanda® (Odevixibat) was approved in the EU for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) aged 6 months or older. Kayfanda is a once-daily, non-systemic ileal bile acid transporter (IBAT) inhibitor that blocks the ileal bile acid transport protein (IBAT), ultimately reducing serum bile acids formed in the liver. Data indicate that patients in the Kayfanda group experienced statistically significant and clinically meaningful improvements in scratching severity from baseline to 6 months, with serum bile acid concentrations also showing a statistically significant reduction at the end of treatment.On September 24, Junshi Biosciences announced that the anti-PD-1 monoclonal antibody drug Toripalimab had recently been approved by the European Commission for two indications: Toripalimab in combination with cisplatin and gemcitabine for the first-line treatment of adult patients with recurrent, non-operable or non-radiation therapy eligible, or metastatic nasopharyngeal carcinoma (NPC); Toripalimab in combination with cisplatin and paclitaxel for the first-line treatment of adult patients with unresectable advanced/recurrent or metastatic esophageal squamous cell carcinoma (ESCC). Study results showed significant efficacy and good safety for both indications, offering patients better survival hope.New Drug Approvals Worldwide (Excluding China) (Partial)
Global New Drug Submission ProgressAccording to the statistical analysis by PharmaDJ, during this statistical period (September 21-27, 2024), there were a total of 6 new drug marketing applications globally (excluding China). Among them, there were 3 NDA submissions, 2 BLA submissions, and 1 vaccine. Compared with the previous statistical period,This increase includes 3 NDA/BLA submissions.On September 25, IntraBio announced that the FDA had accepted the new drug application for IB1001 for the treatment of Niemann-Pick disease type C. The IB1001-301 trial met all primary and key secondary endpoints, improving neurological signs and symptoms, functionality, and quality of life in patients, with the drug being well-tolerated.NDA/BLA Submission (Partial)
According to the data analysis by PharmaDJ, during this statistical period (September 21-27, 2024), a total of five drugs (excluding those in China) received special regulatory designations globally. Among them, there was one chemical drug, three biologics, and one vaccine. Compared with the previous statistical period,This time, two drugs granted special status by regulatory authorities have been added.
On September 23, uniQure NV announced that the FDA had granted AMT-191 Orphan Drug Designation for the treatment of Fabry disease. The Phase I/IIa clinical trial of AMT-191 consists of two cohorts: a low-dose cohort of 6x10 (13GC/kg) and a high-dose cohort of 3x10 (14GC/kg), administered via a single intravenous infusion. Patients will continue to receive conventional enzyme replacement therapy until withdrawal criteria are met and will undergo 24 months of follow-up. The trial will explore early signs of safety, tolerability, and efficacy by measuring the expression of the lysosomal enzyme aGLA-A.On September 25, Elixirgen Therapeutics announced that the FDA had granted EXG-34217 Rare Pediatric Disease Designation (RPDD) for the treatment of patients with dyskeratosis congenita and related telomere biology disorders (DC/TBD). EXG-34217 consists of autologous CD34+ hematopoietic stem cells (HSCs), which are used in EXG-001 ex vivo processing (EXG-001 is a Sendai virus vector). The company is currently recruiting patients at Cincinnati Children's Hospital Medical Center for a Phase I/II trial to evaluate the effects of EXG-34217 in patients with telomere biology disorders.Special Qualification Recognition
Global New Drug Development ProgressAccording to the PharmaDJ data analysis, during this statistical period (September 21-27, 2024), there were a total of 60 updates on the global (excluding China) new drug clinical R&D status, covering 12 fields including oncology, hematology and lymphatic diseases, genetic metabolic disorders, neurological diseases, and immune system diseases.
Among them, the clinical progress updates in the oncology field ranked first among all fields, with 17 entries: involving 9 chemical drugs, 7 biologics, and 1 cell therapy.On September 24, Maiyingnuo Pharmaceuticals announced that its novel skin drug, MDI-1228_mesylate gel, received FDA's implied approval to officially enter Phase II clinical trials for the indication of chronic ulcerative foot caused by diabetes. Preclinical studies have shown that MDI-1228 fundamentally improves wound healing by promoting the generation of adipose-derived stem cells and myofibroblasts at the wound site, which is expected to bring significant benefits to patients with diabetic foot in clinical practice.On September 26, Yilian Biotech registered a Phase III clinical trial on Clinicaltrials.gov for its new B7H3 ADC drug YL201 in the treatment of small cell lung cancer. The trial plans to enroll 438 patients with recurrent small cell lung cancer and is expected to be preliminarily completed by the end of 2027, with OS as the primary endpoint. At the 2024 ESMO conference, Yilian Biotech disclosed the latest clinical data for YL201: As of August 9, 2024, among 276 patients with at least one post-baseline tumor assessment, the ORR was 44.6%, and the DCR was 83.7%. In ES-SCLC patients, the ORR for YL201 was 68.1%, with an mPFS of 6.2 months.Global New Drug Development Progress Details (Partial)
Global Pharmaceutical Transaction EventsThis statistical cycle（2024.09.21-09.27）There were a total of 34 pharmaceutical transaction events globally (including China), involving multiple transactions such as drug rights transfers and company acquisitions.
Summary Table of Global Pharmaceutical Transaction Times (Partial)
Drug Approval/Development Updates in ChinaNew Drug Approvals in ChinaAccording to the data analysis by PharmaDJ, during this statistical period (September 21-27, 2024), a total of 3 new drugs were approved for marketing by the NMPA in China. Among them, 1 was approved via an NDA, 1 received approval for a new indication, and 1 was approved as a new formulation. Compared with the previous statistical period, there are 2 more new drug approvals by the NMPA in this cycle.
On September 25, the latest announcement on the NMPA website showed that the marketing application for Osilodrostat Phosphate Tablets submitted by Ruikangdi Pharmaceuticals has been approved. According to the priority review disclosure by the CDE, the approved indication for this drug is: treatment of endogenous Cushing's syndrome (also known as: endogenous hypercortisolism) in adults. Osilodrostat Phosphate Film-Coated Tablets (Osilodrostat) is a novel oral cortisol synthesis inhibitor, which can control or normalize cortisol levels in adult patients by blocking adrenal cortisol synthesis and inhibiting excessive cortisol production.On September 25, Haihe Biopharma and Daehwa Pharmaceutical jointly announced: The oral paclitaxel solution developed through their collaboration has been approved by the NMPA for marketing in mainland China, indicated for the treatment of patients with advanced gastric cancer whose disease has progressed during or after first-line fluorouracil-based therapy. As of February 15, 2023, the mOS in the trial group was 9.13 months. Oral paclitaxel solution demonstrated superiority over injectable paclitaxel, with a median OS extension of 2.59 months (HR 0.770, 95.5% CI: 0.635, 0.934, p=0.006). In terms of safety, the overall profile was well-tolerated.New Drug Approvals in China (Partial)
Progress of Clinical Implied Consent for New Drugs in ChinaAccording to the data analysis by PharmaDJ, during this statistical period (September 21-27, 2024), a total of 32 new drugs received clinical tacit approval in China, involving 52 application numbers. Among them, there were 22 chemical drugs, 9 therapeutic biological products, and 1 preventive biological product. Compared with the previous statistical period, this time there was a decrease of 8 application numbers for clinical tacit approval.
This Week's Progress on Clinical Trial Approvals for New Drugs in China (Partial)
Progress in New Drug Applications in ChinaAccording to the statistical analysis by PharmaDJ, during this statistical period (September 21-27, 2024), there were a total of 8 new drug marketing applications in China, involving 11 acceptance numbers. Among them, there were 4 chemical drugs and 4 therapeutic biological products. Compared with the previous statistical period, this time saw a reduction of 6 acceptance numbers for new drug marketing applications.
New Drug Applications and Market Launches in China (Partial)
According to the data analysis by PharmaDJ, during this statistical period (September 21-27, 2024), there were 61 new drug clinical trial applications in China, involving 104 acceptance numbers. Among them, there were 25 chemical drugs, 33 therapeutic biological products, and 3 preventive biological products. Compared with the previous statistical period, this period saw an increase of 60 clinical trial application acceptance numbers.
Clinical Trial Applications for New Drugs in China (Partial)
According to the statistical analysis by PharmaDJ, during this statistical period (September 21-27, 2024), a total of six drugs in China received special designation from the NMPA. Among them, three are chemical drugs and three are biologics. Compared with the previous statistical period, four more drugs received special regulatory designations in this cycle.
On September 23, the CDE website announced that the Aficamten Tablets submitted by Jixing Pharmaceuticals are proposed to be included in the priority review, intended for the treatment of symptomatic obstructive hypertrophic cardiomyopathy (oHCM). Preclinical studies have shown that Aficamten can prevent myosin from entering the force-producing state by directly binding to cardiac myosin at a unique and selective allosteric binding site, thereby reducing myocardial contractility. Research results indicate that 24 weeks of Aficamten treatment significantly improved exercise capacity, with patients' peak oxygen uptake (pVO2) measured by cardiopulmonary exercise testing (CPET) increasing by 1.8 mL/kg/min. Additionally, the therapeutic effects of Aficamten were consistent across all predefined subgroups and were well tolerated.On September 25, the CDE website announced that the injectable drug Disitamab Vedotin, submitted by RemeGen, is proposed for priority review. It is intended for HER2-positive (HER2 immunohistochemistry results of 3+ or FISH+) patients with advanced breast cancer and liver metastases who have previously been treated with trastuzumab or its biosimilars and taxane drugs. Study results show that treatment with Disitamab Vedotin resulted in a median RWPFS of 5.9 months and an ORR of 29.6%, including one patient achieving complete remission. In trastuzumab-resistant and refractory patients, RC48 demonstrated median RWPFS of 6.5 months and 5.6 months, respectively.Drugs with Special Qualification Designation by Regulatory Authorities in China (Partial)
Progress in New Drug Development in ChinaAccording to the PharmaDJ data analysis, during this statistical period (September 21-27, 2024), there were a total of 7 updates on the clinical development status of new drugs in China, covering three therapeutic areas: oncology, infectious diseases, and neurological disorders. Among them, 3 were chemical drugs and 4 were biologics. Compared with the previous statistical period, there was a decrease of 4 updates on the clinical development status of new drugs in China.
On September 24, the Phase I clinical trial of RJK002, an AAV gene therapy drug for amyotrophic lateral sclerosis (ALS) independently developed by Ruikang, officially entered the full-speed advancement stage of clinical research. The single-arm IIT study demonstrated that RJK002 has good safety and showed encouraging results in the low-dose and some medium-dose patients whose data have been collected: RJK002 reduced the levels of the biomarker neurofilament light chain (NfL) by nearly 60% after 24 weeks of treatment. In October 2023, RJK002 received Orphan Drug Designation (ODD) from the U.S. FDA.On September 27, at the 2024 CSCO Annual Meeting, Chia Tai Tianqing orally presented the latest results of its Class 1 new drug Cumoxil capsule in a Phase III clinical trial (TQB3616-III-01) for hormone receptor-positive/human epidermal growth factor receptor 2-negative (HR+/HER2-) advanced breast cancer after endocrine therapy: The median progression-free survival (PFS) was 16.62 months, the objective response rate (ORR) was 40.21%, and overall survival (OS) showed a beneficial trend. Its enhanced CDK2 and CDK4 inhibitory activity may help overcome resistance to current CDK4/6 inhibitors in clinical practice.Progress in New Drug Development in China (Partial)
Policy and Regulatory Updates in China's New Drug Development FieldNotice on Publicly Soliciting Opinions for the "Technical Guidelines for Clinical Trials of Thalassemia Gene Therapy Products (Draft for Comments)"In recent years, our center has received multiple communication exchanges and clinical trial applications for thalassemia gene therapy products. To guide and standardize the design of such clinical trials, our center has drafted the "Technical Guiding Principles for Clinical Trials of Thalassemia Gene Therapy Products," which has now been formed into a consultation draft.We sincerely welcome valuable opinions and suggestions from all sectors of society on the draft for comments, and please feedback to us in a timely manner for subsequent improvement. The comment solicitation period is 1 month from the date of publication.Hot News in China's New Drug Development FieldKangfang Biologics, the leader in bispecific antibodies, is about to play its "next ace"
So far, Akeso Biopharma can be regarded as one of the "hottest stocks" in the Hong Kong stock market in 2024.As of the closing on September 25, Akeso Inc. has achieved a cumulative increase of 36.75% this year, with a total market value reaching HKD 54.94 billion. Especially since the announcement at the end of May regarding the head-to-head study where PD-1/VEGF bispecific antibody Ivonescimab outperformed Keytruda (K drug), the stock price has surged significantly, with the highest increase exceeding 120% during this period.Notably, after Kangfang Biotech's remarkable achievements in bispecific antibodies and anti-tumor fields, its innovation story is far from over. The autoimmune pipeline is poised to continue writing a new legend.For more information, please read the original text.Billions of Dollars Worth of Sword Unsheathed? BMS Steps OutA Single Stone Raises a Thousand Ripples.A sharp sword worth tens of billions of dollars in the hands of Bristol-Myers Squibb (BMS) has finally been unsheathed: On September 26, KarXT received FDA approval for market launch, becoming the first newly-mechanized schizophrenia treatment drug approved in decades.KarXT is a potential blockbuster drug that BMS acquired through its $14 billion acquisition of Karuna in December 2023. Evaluate predicts its sales could reach $3.1 billion by 2030.This will undoubtedly be a major turning point for BMS, whose flagship product is facing a patent cliff and is in dire need of new products to fill the gap. More noteworthy is that this is not the only surprise BMS will bring to the market in the future.For more information, please read the original text.These Innovative Drugs Made in China Are About to Be Approved for Market LaunchAlthough the industry is still in a downturn, innovative pharmaceutical companies have achieved good "results." Chinese pharmaceutical companies are continuously making new progress in researching innovative targets and mechanisms, expanding their product pipelines. Recently, which innovative drugs are expected to be approved for marketing? This article reviews the product pipelines and R&D progress of major innovative pharmaceutical companies, selecting innovative drugs that are likely to be approved for marketing in the near future to share with readers.Innovent Biologics: MASH Peptide Weight Loss and Diabetes Indications Have Both Submitted NDAH1 2024 Product Revenue Reached 3.81 Billion Yuan, Increasing by 55% Year-on-Year; Sales Performance of Major Products Such as Sintilimab Continues to Be Strong, Contributing Increasingly to Revenue.Little D Has Something to Say
For the convenience of readers to read and save, we have organized the original weekly report into a PDF version. If you need to obtain the full text, you can click on the blue words at the top.PharmSnap DailyReply "1011 Innovative Drug Weekly" in the official account backstage to download.
