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Milestones · 10 firsts

Drugs that changed medicine.

A curated retrospective, not a feed: first-in-class approvals and practice-changing trial results, whenever they happened. Each entry earns its place by being a genuine first, and links the pivotal trial and the published research behind it.

  1. December 8, 2023First CRISPR/Cas9 gene-editing therapy ever approved by the FDA

    The first medicine built by directly editing the human genome

    Exagamglogene autotemcel · Casgevy

    Casgevy doesn't work like a normal medicine you keep taking — it's a one-time treatment that directly rewrites a small piece of a patient's own DNA. Doctors take out a patient's bone marrow stem cells, use the gene-editing tool CRISPR to snip out a specific genetic switch, and put the edited cells back in. That switch normally keeps a backup form of hemoglobin turned off after infancy; flipping it back on gives sickle cell patients a working alternative to the sickle-shaped hemoglobin causing their disease. It was the first time the FDA had ever approved a treatment built this way — editing a gene directly, rather than giving a drug that acts on the body from outside.

  2. 5 months earlier

    July 6, 2023First Alzheimer's drug to win full FDA approval with confirmed clinical benefit on cognitive decline

    The first Alzheimer's drug proven, not just hoped, to slow the disease

    Lecanemab · Leqembi

    For about twenty years, drug after drug aimed at Alzheimer's disease failed to actually slow it down in a real, confirmed way, even when they cleared the sticky amyloid plaques the disease is known for. Lecanemab was the first to break that streak: in a large trial, patients taking it experienced measurably slower decline in memory and daily function than patients on placebo, not just less plaque on a brain scan. That's why the FDA gave it full, traditional approval rather than the narrower, evidence-still-pending approval an earlier amyloid drug had received — it's the first Alzheimer's treatment the FDA has confirmed actually does what patients and families hope it will.

  3. 8 months earlier

    November 17, 2022First drug ever approved to delay the onset of type 1 diabetes, not just treat it

    The first drug that delays a disease before it fully starts

    Teplizumab · Tzield

    Type 1 diabetes doesn't appear overnight — there's often a silent stage beforehand where the immune system is already attacking the pancreas, detectable by blood tests, before a person has any symptoms. Teplizumab was the first drug ever approved to intervene at that earlier stage: a two-week course of infusions calms down the immune attack enough to buy patients real time, an extra two years on average, before they need insulin. It doesn't cure or fully prevent the disease, but it was the first drug of any kind shown to delay a disease's onset in this way, not just treat it once it arrives.

  4. 5 years earlier

    August 30, 2017First CAR-T cell therapy, and first gene therapy of any kind, ever approved by the FDA

    The first approved cell therapy engineered from a patient's own immune system

    Tisagenlecleucel · Kymriah

    Kymriah wasn't just a new drug — it was a new kind of medicine entirely. Instead of giving a patient a manufactured compound, doctors take a patient's own immune cells out of their body, genetically reprogram them in a lab to recognize and attack their specific leukemia, and infuse them back in. It was the first therapy like this the FDA had ever approved for anyone, for any disease — the first "living drug." It opened the door for the wave of cell and gene therapies that followed, including Casgevy, also on this site, the first CRISPR-based treatment ever approved.

  5. 3 years earlier

    December 3, 2014First bispecific T-cell engager (BiTE) antibody ever approved

    The antibody engineered to physically wire a T cell to its target

    Blinatumomab · Blincyto

    Most antibody drugs have two identical arms that grab the same target. Blinatumomab is built differently: one arm grabs a marker on leukemia cells, and the other arm grabs a killer T cell, physically wiring the two together so the T cell attacks the cancer cell it's now stuck to. It was the first drug of this two-armed "bispecific" design ever approved, and the engineering trick it pioneered, forcing an immune cell into direct contact with a cancer cell, has since been copied by many other cancer drugs.

  6. 3 years earlier

    January 31, 2012First drug to treat the underlying genetic cause of cystic fibrosis, not just its symptoms

    The first cystic fibrosis drug to fix the broken protein, not just the symptoms

    Ivacaftor · Kalydeco

    Cystic fibrosis is caused by a broken chloride channel in cells, which leads to thick mucus, lung infections, and other symptoms doctors used to treat one by one. Ivacaftor was the first CF drug to instead go after the actual broken part: for patients with a specific version of the defect (the G551D mutation), it helps the faulty channel open more normally, treating the root cause rather than just the downstream problems it causes. It only works for the roughly 4-5% of CF patients with this particular mutation, but it proved that fixing the gene's protein directly was possible — a breakthrough that later led to combination drugs covering most people with CF.

  7. 10 months earlier

    March 25, 2011First immune checkpoint inhibitor ever approved

    The drug that proved the immune system itself could be a cancer treatment

    Ipilimumab · Yervoy

    Cancer cells often survive by tricking the immune system into leaving them alone, using built-in "off switches" on immune cells. Ipilimumab was the first drug ever approved that works by disabling one of those switches (CTLA-4) rather than attacking the tumor directly — it frees the immune system to recognize and fight the cancer itself. It was the first drug of any kind shown to help people with advanced melanoma live longer in a randomized trial, and it proved that unleashing the immune system was a viable cancer treatment strategy, opening the door to later checkpoint drugs like pembrolizumab and nivolumab, also on this site, which target a related but distinct brake called PD-1.

  8. 10 years earlier

    May 10, 2001First BCR-ABL tyrosine kinase inhibitor

    The drug that launched targeted cancer therapy

    Imatinib · Gleevec

    Before imatinib, cancer drugs mostly worked by poisoning any fast-growing cell, tumor and healthy tissue alike. Imatinib was different: researchers had identified the exact broken protein driving chronic myeloid leukemia, a fusion of two genes called BCR-ABL, and imatinib was built specifically to jam it. The results were dramatic — a disease that used to kill most patients within a few years became one people could live with for decades. Its success proved that hunting down a cancer's specific molecular cause, rather than just attacking anything that divides quickly, could actually work — an approach the whole field now calls targeted therapy.

  9. 3 years earlier

    March 27, 1998First oral treatment for erectile dysfunction, and the first PDE5 inhibitor ever approved

    The heart drug that failed at its job and succeeded at another

    Sildenafil · Viagra

    Viagra started out as a failed heart drug. Pfizer was testing it for chest pain and high blood pressure, but it didn't work especially well for that — what it did do, unexpectedly, was cause erections in some of the male trial participants, a side effect researchers noticed and decided to pursue on its own. Repurposed and approved in 1998, it became the first pill ever approved for erectile dysfunction, replacing injections and implants as the standard treatment, and it launched an entire class of drugs, called PDE5 inhibitors, that includes tadalafil, also on this site.

  10. 4 months earlier

    November 26, 1997First monoclonal antibody ever approved for cancer treatment

    The antibody that opened the era of biologic cancer therapy

    Rituximab · Rituxan

    Rituximab was the first lab-made antibody ever approved to treat cancer. It works by recognizing a marker called CD20 on the surface of B cells, including the cancerous ones in certain lymphomas, and flagging them for destruction by the immune system. Before this, cancer antibodies were mostly experimental; Rituximab proved the concept could work as an actual approved treatment, and it opened the door to the wave of antibody-based cancer and immune drugs that followed, including trastuzumab and pembrolizumab, both also on this site.