Two Chinese Orphan Drugs Ranked Among Global Top 10 Most Valuable by 2028: Carvykti and Brukinsa Lead Domestic Innovation

Vertex began exploring the cystic fibrosis (CF) field as early as 2001. In 2019, the cystic fibrosis transmembrane conductance regulator (CFTR) modulator Trikafta (elexacaftor, tezacaftor, and ivacaftor) was approved by the FDA for marketing.Becoming the First CF Triple Combination Drug。Trikafta directly targets the CFTR protein, forming a striking competitive advantage over CF drugs available on the market at the time that could only alleviate symptoms, thereby monopolizing nearly 90% of the CF market.

Once launched, Trikafta quickly became a blockbuster drug, generating $8.945 billion in sales for Vertex last year.Expected to generate $8.753 billion in revenue by 2028.

On July 2, the U.S. FDA accepted Vertex's New Drug Application (NDA) for its next-generation triple combination therapy (vanzacaftor/tezacaftor/deutivacaftor, referred to as vanza triple therapy) and granted it priority review, shortening the review cycle from the original 10 months to 6 months. The PDUFA date is January 2, 2025.Vanza triple therapy has the potential to become a blockbuster drug and even surpass Trikafta in sales.

Hemlibra (Emicizumab) is a bispecific antibody targeting coagulation factor IXa and coagulation factor X.For the treatment of Hemophilia A.HemlibraIt is a preventive treatment drug that can be administered via a ready-to-use solution for subcutaneous injection. Hemlibra was developed by Chugai Pharmaceutical Co., Ltd. and is being co-developed globally by Chugai Pharmaceutical, Roche, and Genentech.

In November 2017, Hemlibra was approved by the FDA for marketing to prevent or reduce the frequency of bleeding in adult and pediatric patients with factor VIII inhibitors in hemophilia A. In April 2018, Hemlibra received a breakthrough therapy designation from the FDA for an expanded new indication for the treatment of hemophilia A without factor VIII inhibitors.This bispecific antibody drug is currently the only one applied in the field of hemophilia.

Since its launch, Hemlibra has achieved an annual compound growth rate of over 300%, surpassing the $2 billion sales milestone in 2020 to become a blockbuster product. Hemlibra's 2023 sales reached 4.147 billion Swiss francs (approximately...$4.575 billion, an increase of 16% year-on-year. The growth trend continues into 2024.Sales in the first half of 2024 were 2.143 billion Swiss francs (approximately 2.529 billion US dollars)Sales are expected to reach $6.203 billion by 2028.

Ravulizumab is a long-acting complement C5 inhibitor, which was first approved by the FDA for marketing in December 2018.For the treatment of paroxysmal nocturnal hemoglobinuria (PNH)The trade name is Ultomiris. In September 2021, ravulizumab received FDA approval for a new indication to treat adult and pediatric (over one month old) patients with atypical hemolytic uremic syndrome (aHUS). In April 2022, the third indication for ravulizumab was approved by the FDA, for the treatment of adult patients with generalized myasthenia gravis (gMG).

Developed through the collaboration between Legend and Johnson & JohnsonCarvyktiIt is a gene-modified autologous T-cell immunotherapy targeting BCMA.In December 2017, Janssen Pharmaceuticals, a Johnson & Johnson company (now Johnson & Johnson Innovative Medicine), entered into a global exclusive license and collaboration agreement with Legend Biotech to develop and commercializeCilta-celIn February 2022, Carvykti was approved by the FDA for marketing, indicated for the treatment of relapsed or refractory patients who have previously received at least four lines of therapy.Multiple MyelomaAdult patients.

Carvykti generated sales of $133 million in its first year on the market, and sales surged 276% to $500 million in 2023.H1 2024 Sales Reached $343 Million, Increasing by 81% Year-over-Year.Sales are expected to reach $4.406 billion by 2028.

On August 27, according to the latest announcement on the official website of China's NMPA, the new drug application for Cilta-cel Injection submitted by Legend Biotech has been officially approved. According to the priority review announcement from the CDE, the indication approved this time is: for the treatment of adult patients with relapsed or refractory multiple myeloma (R/R MM) who have previously received treatment with one proteasome inhibitor and one immunomodulatory agent., isThe 6th CAR-T therapy approved in China.

RucotideNi isIncyteOpenA small molecule JAK1/JAK2 inhibitor, its tablet (brand name: Jakafi) was approved for marketing in the United States in December 2011, and its cream (brand name:Opzelura) was approved for marketing in the United States in September 2021. The tablets are indicated for polycythemia vera, myelofibrosis, and graft-versus-host disease, while the cream is indicated for atopic dermatitis and non-segmental vitiligo.

Incyte's Total Revenue in H1 2024 was $1.925 billion, a year-over-year increase of 9%. The company’s most important product, the JAK1/2 inhibitor ruxolitinib, accounted for over 90% of total product sales. Jakafi sales reached $1.278 billion, up 1% year-over-year, while Opzelura sales were $207 million, marking a 52% year-over-year increase. Incyte forecasts Jakafi sales to grow from $2.59 billion in 2023 to between $2.69 billion and $2.75 billion in 2024. Evaluate projects the product’s sales to reach $4.235 billion by 2028.

Calquence (Acalabrutinib)Is a new generation of BTK selective inhibitor, throughBTK Covalent BindingInhibit its activity.In 2016, AstraZeneca acquired 55% of Acerta Pharma for $4 billion.

CalquenceIt was previously granted Priority Review, Breakthrough Therapy designation, and Orphan Drug designation by the U.S. FDA. In 2017, it received accelerated approval from the FDA for the treatment of relapsed mantle cell lymphoma (MCL). It was later approved for use as an initial therapy or subsequent treatment for adult patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).

March 2023, China NMPA approves acalabrutinib capsules (trade name: Calquence/Kang Ke Qi)For the first timeLaunched. For the treatment of adult patients with mantle cell lymphoma (MCL) who have received at least one prior therapy.August 2023, as a single agent, is indicated for adult patients with chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL) who have received at least one prior therapy.Item 2Indications approved in China;August 28, 2024, The latest announcement on the CDE official website in China shows that AstraZeneca has submittedAcalabrutinib (Generic Name: Acalabrutinib Capsules, Brand Name: Kankeqi)The listing application has been accepted:It is speculated that the new indication for Acalabrutinib's marketing application this time may be first-line treatment.Chronic Lymphocytic Leukemia（CLL）。

As of 2023, the global sales of Calquence have reached $2.514 billion, with global sales of $1.508 billion in H1 2024. It is projected that by 2028, sales will grow to $4.016 billion, reflecting its ongoing growth potential and commercial prospects in the treatment of B-cell malignancies.

Brukinsa (Zanubrutinib) is a potent BTK inhibitor, used as a monotherapy or in combination with other therapies. It is mainly used in the clinical treatment of adult T-cell lymphoma, adult acute lymphoblastic leukemia, or small lymphocytic lymphoma.

Lynparza(Olaparib, Lynparza)It is an oral PARP inhibitor that disrupts DNA repair in cancer cells, leading to cancer cell death. The drug has been approved for the treatment of ovarian cancer, fallopian tube cancer, or primary peritoneal cancer, as well as breast cancer, pancreatic cancer, and prostate cancer. Olaparib was developed by KuDOS Pharmaceuticals in the UK and later acquired by AstraZeneca. It was first approved in the US in 2017, imported into China in 2018, and included in the National Reimbursement Drug List in 2019.

2023LynparzaSales were $2.811 billion, with H1 2024 sales reaching $1.45 billion, a year-over-year increase of 9%.Predicted sales of the drug in 2028: $3.451 billion.

Its compound patent has expired this year.Expires in March, formulation patent expires in October 2029,The first domestic manufacturer to launch a generic version of Olaparib in China is Qilu Pharmaceutical, securing the first-to-market position; Kelun Pharmaceutical's Olaparib tablets are the second domestically produced version in China.In addition, multiple companies such as Shancor Pharmaceutical, Nanjing Fosun Pharmaceutical Technology, Huadong Pharmaceutical, CSPC Pharmaceutical Group, and Xentai Pharmaceutical have also joined the intense competition.

Venclexta is a "first-in-class" BCL-2 inhibitor. The BCL-2 protein is known to be overexpressed in blood cancers, mediating tumor survival and treatment resistance.Venetoclax was initially2016Year obtainedFDAApproval for the treatment of existing17pMissing Chronic Lymphocytic Leukemia（CLL）。In 2018, the U.S. FDA approved the expansion of its indications toWhether or not17pPatients with relapsed/refractory CLL who have previously received at least one therapy can use this drug.

Venclexta has maintained a strong growth momentum in recent years, reaching $2.288 billion in 2023, a year-on-year increase of 13.9%. Venclexta (Venetoclax) showed steady growth, with sales in the first half of the year reaching $1.251 billion, a year-on-year increase of 16%.Predicted sales of the drug to reach $3.344 billion by 2028.