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There are more than 7,000 known rare diseases worldwide, affecting approximately 400 million patients
.
80% of these diseases are caused by genes, and 50% affect children
.
In summary, patients with rare diseases represent one of the world's largest groups of medically unmet patients, with only 5% of known therapies for rare diseases having one or more therapies approved
.
In recent years, rare diseases have also become a new therapeutic field for pharmaceutical giants to compete for, and Pfizer's layout has attracted much attention
.
Rare disease revenue was $3.
5 billion
5 billion
As one of Pfizer's six therapeutic areas of the drug business (others include oncology, inflammation and immunology, hospitals, vaccines and internal medicine), Pfizer's current portfolio spans four therapeutic areas, including: rare hematology, rare endocrinology/metabolism, rare neurology and rare cardiology
.
Pfizer currently has approved products for amyloidosis, hemophilia and endocrine diseases
.
In terms of the overall Pfizer rare disease drug market, the proportion of the company's total revenue (excluding new crown vaccines and drugs) has continued to grow, from 4% in 2017 to 8%
in 2021.
Among them, in 2019, the treatment of transthyretin amyloidosis (ATTR) cardiomyopathy and polyneuropathy drugs Vyndaqel (clobenzolate glucosamine) / Vyndamax (clobenzole acid) was approved by the FDA in the United States, officially opening the Pfizer rare disease drug market growth mode
again.
Vyndaqel/Vyndamax reached a bombshell level ($1.
288 billion) in 2020 and $2.
015 billion in 2021 (accounting for 57% of Pfizer's total rare disease drug revenue that year).
Meanwhile, Pfizer's total revenue from rare disease drugs has soared from about $2.
2 billion per year from 2017-2019 to $3.
5 billion in 2021, an increase of 58%.
In addition, Pfizer's hemophilia B drug BeneFIX (recombinant human coagulation factor IX.
), human growth hormone replacement therapy Genotropin (recombinant human growth hormone), and hemophilia type A drug Refacto
AF (Moro coagulin α)/Xyntha (recombinant human coagulation factor VIII) and acromegaly drug Somavert (pegvisomant) have been selling hundreds of millions of dollars
for many years.
The future bets on gene therapy
The future bets on gene therapyToday, Pfizer is working to lay out a new generation of breakthrough therapies, including several models: small molecules, large molecules/monoclonal antibodies (mAbs), gene therapies, and even devices, hoping to regain a high ground in the field of rare diseases through potential new therapies, and the current standard of treatment is far from sufficient
for the population of patients with multiple rare diseases.
Pfizer's deep pipeline includes more than 10 investigational drugs in clinical development, and more than 5 new molecular entities and 3 gene therapy programs have entered Phase 3 clinical trials
.
80% of rare diseases are hereditary, so Pfizer is exploring a new potentially transformative approach to treating genetic diseases
through gene therapy.
Recombinant adeno-associated virus (rAAV) precisely targeted gene therapy for continuous cell-specific therapy
.
Currently, Pfizer prioritizes single-gene defective diseases such as Duchenne muscular dystrophy (DMD), hemophilia, and amyotrophic lateral sclerosis (ALS
).
Pfizer has a strong pipeline of potential gene therapies, with 3 therapies in Phase 3 clinical trials
.
Pfizer hopes to apply gene therapy to treat more common and complex diseases in the future, which involve multiple genes, such as central nervous system diseases and heart disease
.
With 2016 on Bamboo
With the acquisition of Therapeutics, Pfizer expands its portfolio to advance recombinant AAV-based gene therapy technologies, and Pfizer's first gene therapy candidate enters clinical development: the gene therapy PF-06939926 for the treatment of DMD initiates a Phase 3 clinical trial
in 2021.
Another partner, Spark Therapeutics, will treat hemophilia B with the investigational gene therapy fidanacogene
Following the transfer of Elaparvovec to Pfizer, a pivotal Phase 3 clinical trial
has also been initiated.
Pfizer also works with Sangamo
Therapeutics has entered into an exclusive global collaboration and license agreement to develop and commercialize gene therapy programs, which currently include ongoing Phase 1/2 trials for hemophilia A, and preclinical programs
for amyotrophic lateral sclerosis (ALS).
。 In Giroctocogene
Following the transfer of the IND of fitelparvovec (formerly SB-525, now PF-07055480), Pfizer initiated an evaluation of giroctocogene
A Phase 3 trial
of fitelparvovec gene therapy for severe hemophilia A.
