Inventory of blockbuster innovative medicines listed globally in 2019

Release date: 2020-01-14 Views: 0

Source: Firestone Creation  

The progress bar in 2019 has finally been updated to 100%. Looking back on this year, there have been many heavy-duty innovative drugs listed globally, especially in the field of tumor-targeted drugs, such as the world's first FGFR inhibitor, the first breast cancer PI3K inhibitor, etc. It has been on the market successively, adding many guns and ammunition to humans against cancer. In addition, gene therapies such as Zolgensma are also available in 2019.


First new mechanism antidepressant in 30 years

Common name: Esketamine

Product Name: Spravato

Original researcher: Johnson & Johnson

Time to market: FDA-March 5, 2019

Indication: Adults with major depression (TRD) who develop resistance

The FDA-approved Spravato (esketamine) CIII nasal spray in March is a non-competitive antagonist of the N-methyl-D-aspartate (NMDA) receptor of Johnson & Johnson's Janssen Pharmaceuticals, combined with oral antidepressants For the treatment of adult patients with refractory depression (TRD).

Compared with standard oral therapies, Spravato has the advantage of rapid onset by intranasal administration. This is also the first antidepressant with a new mechanism of action in more than 30 years. The drug has been approved by the US FDA for two breakthrough therapies for the treatment of drug-resistant depression and major depression with impending suicide risk. An application for a new drug was submitted to the EMA in October last year; in China, the drug is in the clinical application stage and has entered a special review channel.


First oral medication for active secondary progressive multiple sclerosis (SPMS)

Common name: Siponimod

Product Name: Mayzent

Original researcher: Novartis

Time to market: FDA-March 26, 2019

Indications: For the treatment of adult patients with relapsing multiple sclerosis (MS), including active secondary progressive multiple sclerosis (SPMS), relapsing-remitting multiple sclerosis (RRMS), and clinically isolated syndrome (CIS) )

In March 2019, the FDA approved the marketing of Mayzent (siponimod) developed by Novartis for the treatment of adult patients with relapsing multiple sclerosis (RMS), including active secondary progressive disease (active SPMS), relapsing-remitting disease (RRMS) and Clinically Isolated Syndrome (CIS).

Novartis also submitted an application to the EMA in October 2018. Siponimod is a highly selective regulator of S1P receptor specific isoforms and can selectively bind to S1P1 and S1P5 receptor subtypes. When combined with the S1P1 receptor subtype expressed on the surface of lymphocytes, it prevents lymphocytes from entering the central nervous system (CNS) in patients with MS, thereby reducing the inflammatory response.

Siponimod is an upgraded product of Novartis blockbuster Gilenya after FDA safety warnings in 2018. For multiple sclerosis (MS), the drug breaks the situation where there is no medicine available for patients with active secondary progressive multiple sclerosis (SPMS).


World's first FGFR inhibitor

Common name: Erdafitnib

Product Name: Balversa

Original researcher: Johnson & Johnson

Time to market: FDA-April 12, 2019

Indications: For patients with locally advanced or metastatic urothelial carcinoma (UC) who have progressed after chemotherapy and whose tumor has specific fibroblast growth factor (FGFR) gene changes

In April 2019, the FDA accelerated the approval of Balversa (Erdafitinib) of Johnson & Johnson's Janssen Pharmaceutical Company for adults with locally advanced or metastatic urothelial carcinoma (mUC) carrying specific fibroblast growth factor receptor (FGFR) gene changes patient.

Eraditnib is an oral pan-fibroblast growth factor receptor (FGFR) tyrosine kinase inhibitor. It is also the first FGFR kinase inhibitor approved by the FDA. It received breakthrough breakthroughs from the FDA in March and September 2018, respectively. Drug eligibility (BTD) and priority drug eligibility. In 2008, Johnson & Johnson and Erdafitnib finder Astex Pharmaceuticals signed an exclusive global licensing and cooperation agreement to jointly develop and commercialize Erdafitinib. Approved at the same time as Erdafitnib, there is a companion diagnostic device developed by Qiagen to detect mutations in FGFR2 and FGFR3.


The third IL-23 targeted drug in the world

Common name: Risankizumab

Product Name: Skyrizi

Original researcher: AbbVie

Time to market: PDMA (Japan)-March 26, 2019;

FDA (United States)-April 23, 2019;

EMA (European Union)-April 26, 2019

Indication: Moderate or severe plaque psoriasis

On March 26, 2019, Risankizumab was approved by PMDA to be the first in Japan to be marketed for the treatment of psoriasis vulgaris, arthropathy psoriasis, pustular psoriasis, and erythrodermic psoriasis. . Subsequently, the FDA and EMA approved the drug for marketing in the United States and European Union on April 23, 2019 and April 26, 2019, respectively, for the treatment of adult patients with moderate to severe psoriasis.

Risankizumab is an anti-IL-23 monoclonal antibody drug. It was originally developed by the German pharmaceutical company Boehringer Ingelheim (BI). AbbVie paid a $ 600 million prepayment in February 2016 to obtain the global value of risankizumab. Commercialization rights. This is also the third monoclonal antibody that targets the IL-23p19 subunit after the successful launch of Guselkumab of Jansen and Tildrakizumab of Merck / Sun Pharmaceuticals / Almirall.

On February 14, 2019, "Risankizumab Injection" (ABBV-066), jointly declared by AbbVie and Boehringer Ingelheim, was clinically approved by the State Food and Drug Administration in China for adult active psoriatic arthritis.


First breast cancer PI3K inhibitor

Common name: Alpelisib

Product Name: Piqray

Original researcher: Novartis

Time to market: FDA-May 24, 2019

Indications: PI3K mutations in HR + and HER2- for disease progression after endocrine therapy in combination with fulvestrant for postmenopausal women with breast cancer

On May 24, 2019, the FDA approved Novartis's Piqray (alpelisib) for marketing, which is the first and currently the only approved targeted drug specifically for this part of breast cancer patients. Alpelisib, as the second FDA innovative therapeutic drug obtained by Novartis in 2019, has enriched the product pipeline in the field of anticancer therapy, opened the door to clinical genomics research of breast cancer, and opened a new era of breast cancer molecular typing.


First SMA gene therapy

Common name: Onasemnogene abeparvovec-xioi

Product Name: Zolgensma

Original researcher: Novartis

Time to market: FDA-May 24, 2019

Indications: Used to treat children with spinal muscular atrophy (SMA) caused by an allele mutation of the motor neuron survival gene 1 under 2 years of age

On May 24, 2019, the FDA approved Onasemnogene abeparvovec for marketing in the United States, an adeno-associated virus type 9 vector-based gene therapy for the treatment of spinal cord caused by allele mutations in motor neuron survival gene 1 under 2 years of age Children with muscular dystrophy.

Zolgensma is a gene therapy that uses the AAV9 virus in the NAV® gene delivery technology platform of Regenxbio to deliver the AAV9 vector containing the SMN gene directly to the central nervous system motor neurons through the blood-brain barrier to increase the expression of SMN protein in the body. The FDA has granted related qualifications such as fast track, breakthrough therapy, rare pediatric disease treatment drugs, and priority review, reducing the review time to 8 years.

In January 2018, RegenxBio and AveXis expanded and revised the SMA development and commercialization license agreement. AveXis was granted the exclusive global authorization of the NAV® platform and paid high annual fees and business milestone fees. In April of the same year, Novartis acquired AveXis for a total price of $ 8.7 billion. This is also doomed to Zolgensma's high price, with $ 2.125 million becoming the most expensive drug in history.


First chemotherapy immunotherapy

Common name: Polatuzumab vedotin

Product Name: Polivy

Original researcher: Roche

Time to market: June 10, 2019

Indication: With chemotherapy bendamustine and rituximab products for patients with relapsed or refractory diffuse large B-cell lymphoma (R / RDLBCL)

In June 2019, the FDA accelerated the approval of Roche's Genentech antibody drug conjugate (ADC) Polivuz (polatuzumab vedotin-piiq) in combination with bendamustine and rituximab for at least 2 previously accepted This therapy is for patients with relapsed or refractory diffuse large B-cell lymphoma (R / RDLBCL).

In the United States and the European Union, polatuzumab vedotin has been granted orphan drug status for DLBCL, and has been awarded breakthrough drug status (BTD) and priority drug status (PRIME). Polatuzumab vedotin is a pioneering anti-CD79b humanized fusion protein, which is an anti-CD79b antibody conjugated with the anti-mitotic agent MMAE (monomethylastatin E). It specifically binds to the CD79b protein on B cells and kills cancer cells.


Broad-spectrum anticancer drug nova

Common name: Entrectinib

Product Name: Rozlytrek

Original researcher: Roche

Time to market: August 15, 2019

Indications: Treatment of patients with neurotrophic tyrosine receptor kinase (NTRK) fusion-positive solid tumors, including pancreas, thyroid, salivary, breast, colorectal, and lung cancer.

On August 15, 2019, the FDA accelerated the approval of the world's third broad-spectrum anticancer drug Entrectinib for the treatment of NTRK fusion-positive adults and children, the progression of locally advanced or metastatic solid tumors after initial treatment, or no standard treatment Patients with solid tumors and ROS1-positive non-small cell lung cancer (NSCLC).

Entrectinib is a selective oral NTRK inhibitor developed by biopharmaceutical company Ignyta. At the end of 2017, Roche spent 1.7 billion to acquire Ignyta to obtain the drug. It is also the third "unlimited cancer" drug approved by the FDA after Keytruda and Vitrakvi. Cancer therapy, "unlimited cancer species" refers to the key driver genes that do not target the primary tumor type but only the tumor.

In 2017, Entrectinib has been awarded Breakthrough Therapy BTD, Priority Drug, and Pioneer Drug by the FDA, EMA, and the Japanese Health Department respectively. In June 2019, Japan has approved its use for patients with NTRK gene fusion tumors.


China's first FDA breakthrough therapy

Common name: Zanubrutinib

Product Name: Brukinsa

Original researcher: BeiGene

Time to market: November 14, 2019

Indication: Adult mantle cell lymphoma (MCL) patients who have previously received at least one therapy

On November 14, 2019, the FDA accelerated the approval of Beukinsa (Zanubrutinib, Zebutinib), a BTK inhibitor from BeiGene, for the treatment of adult mantle cell lymphoma (MCL) patients who have previously received at least one therapy. This is the third BTK marketed after Ibrutinib and Acalabrutinib jointly developed by Johnson & Johnson and AbbVie. As the second generation of BTK inhibitors, Zebutinib It has obtained three FDA orphan drug qualifications and breakthrough therapy certifications, and is a fast-track qualification for Fahrenheit macroglobulinemia. It is the first anti-cancer drug independently developed in China and obtained FDA breakthrough therapy certifications and marketed.

It is worth noting that on November 1, BeiGene and Amgen announced that they had reached a strategic global tumor partnership. Amgen purchased 20.5% of BeiGene's shares with approximately US $ 2.7 billion in cash, accelerating the development of global anti-tumor drugs.

L entiGlobin

World's first beta thalassemia gene therapy

Common name: LentiGlobin

Product Name: Zynteglo

Original researcher: Bluebird

Time to market: June 14, 2019

Indications: Transfusion-dependent β-thalassemia (TDT) for non-β0 / β0 genotypes in patients 12 years and older

In June 2019, the European Commission (EC) announced that BluebirdBio's Zynteglo gene therapy successfully passed the European Medicines Agency (EMA) 's rapid evaluation of advanced therapeutic drugs (ATMP) and approved its listing on the European market. This is the world's first gene therapy for transfusion-dependent beta thalassemia.

Zynteglo is a one-time gene therapy using lentivirus as a vector. It collects the patient's own hematopoietic stem cells and adds a functional copy of the modified β-globin gene (βA-T87Q-globin gene) to the patient's own hematopoietic (Blood) stem cells (HSCs), and then reconstitute the transformed cells back into the patient to achieve a potential cure for the disease. The patient offers the possibility of no need for blood transfusion, and the effect is expected to last a lifetime. The FDA awarded LentiGlobin as an orphan drug and breakthrough therapy designation for the treatment of transfusion-dependent β-thalassemia patients (TDT). In addition, LentiGlobin has also obtained EMA's orphan drug qualification, fast track qualification, priority drug qualification (PRIME), and accelerated review qualification in the European Union.

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